User login
Vaping-related lung disease cases rise, case reporting standardized
The number of possible cases of vaping-related pulmonary illness has risen to 215, reported from 25 states, as of Aug. 27, 2019, according to the Centers for Disease Control and Prevention, Atlanta. Additional reports of pulmonary illness are under investigation.
The CDC has released a standardized case definition that states are using to complete their own investigations and verifications of cases. It appears that all cases are linked to e-cigarette product use, but the cause of the respiratory illnesses is still unconfirmed.
In many cases, patients reported a gradual start of symptoms, including breathing difficulty, shortness of breath, and/or chest pain before hospitalization. Some cases reported mild to moderate gastrointestinal illness including vomiting and diarrhea, or other symptoms such as fevers or fatigue. In many cases, patients have also acknowledged recent use of tetrahydrocannabinol (THC)-containing e-cigarette products while speaking to health care personnel or in follow-up interviews by health department staff, according to a statement from the CDC and the Food and Drug Administration.
The agencies are working with state health departments to standardize information collection at the state level to help build a more comprehensive picture of these incidents, including the brand and types of e-cigarette products, whether any of them would fall within the FDA’s regulatory authority, where they were obtained, and whether there is a link to specific devices, ingredients, or contaminants in the devices or substances associated with e-cigarette product use.
CDC staff have been deployed to Illinois and Wisconsin to assist their state health departments. The agencies have released a Clinician Outreach and Communication Activity (COCA) Clinical Action Alert describing this investigation and asking providers to report possible cases to their state health departments. In addition to a standardized case definition, the agencies have issued a medical chart abstraction form and case interview questionnaire, are reviewing and providing feedback on data collection and health messaging tools for states, and are facilitating information sharing between states with possible cases.
More information on the cases and reporting are available from the CDC.
The number of possible cases of vaping-related pulmonary illness has risen to 215, reported from 25 states, as of Aug. 27, 2019, according to the Centers for Disease Control and Prevention, Atlanta. Additional reports of pulmonary illness are under investigation.
The CDC has released a standardized case definition that states are using to complete their own investigations and verifications of cases. It appears that all cases are linked to e-cigarette product use, but the cause of the respiratory illnesses is still unconfirmed.
In many cases, patients reported a gradual start of symptoms, including breathing difficulty, shortness of breath, and/or chest pain before hospitalization. Some cases reported mild to moderate gastrointestinal illness including vomiting and diarrhea, or other symptoms such as fevers or fatigue. In many cases, patients have also acknowledged recent use of tetrahydrocannabinol (THC)-containing e-cigarette products while speaking to health care personnel or in follow-up interviews by health department staff, according to a statement from the CDC and the Food and Drug Administration.
The agencies are working with state health departments to standardize information collection at the state level to help build a more comprehensive picture of these incidents, including the brand and types of e-cigarette products, whether any of them would fall within the FDA’s regulatory authority, where they were obtained, and whether there is a link to specific devices, ingredients, or contaminants in the devices or substances associated with e-cigarette product use.
CDC staff have been deployed to Illinois and Wisconsin to assist their state health departments. The agencies have released a Clinician Outreach and Communication Activity (COCA) Clinical Action Alert describing this investigation and asking providers to report possible cases to their state health departments. In addition to a standardized case definition, the agencies have issued a medical chart abstraction form and case interview questionnaire, are reviewing and providing feedback on data collection and health messaging tools for states, and are facilitating information sharing between states with possible cases.
More information on the cases and reporting are available from the CDC.
The number of possible cases of vaping-related pulmonary illness has risen to 215, reported from 25 states, as of Aug. 27, 2019, according to the Centers for Disease Control and Prevention, Atlanta. Additional reports of pulmonary illness are under investigation.
The CDC has released a standardized case definition that states are using to complete their own investigations and verifications of cases. It appears that all cases are linked to e-cigarette product use, but the cause of the respiratory illnesses is still unconfirmed.
In many cases, patients reported a gradual start of symptoms, including breathing difficulty, shortness of breath, and/or chest pain before hospitalization. Some cases reported mild to moderate gastrointestinal illness including vomiting and diarrhea, or other symptoms such as fevers or fatigue. In many cases, patients have also acknowledged recent use of tetrahydrocannabinol (THC)-containing e-cigarette products while speaking to health care personnel or in follow-up interviews by health department staff, according to a statement from the CDC and the Food and Drug Administration.
The agencies are working with state health departments to standardize information collection at the state level to help build a more comprehensive picture of these incidents, including the brand and types of e-cigarette products, whether any of them would fall within the FDA’s regulatory authority, where they were obtained, and whether there is a link to specific devices, ingredients, or contaminants in the devices or substances associated with e-cigarette product use.
CDC staff have been deployed to Illinois and Wisconsin to assist their state health departments. The agencies have released a Clinician Outreach and Communication Activity (COCA) Clinical Action Alert describing this investigation and asking providers to report possible cases to their state health departments. In addition to a standardized case definition, the agencies have issued a medical chart abstraction form and case interview questionnaire, are reviewing and providing feedback on data collection and health messaging tools for states, and are facilitating information sharing between states with possible cases.
More information on the cases and reporting are available from the CDC.
Rozlytrek approved for ROS1-positive metastatic NSCLC, cancers with NTRK gene fusion defects
Rozlytrek (entrectinib) has been approved to treat cancers with neurotrophic tyrosine receptor kinase (NTRK) gene fusion defects in adults and adolescents for whom there are no effective treatments, the Food and Drug Administration announced in a press release.
Entrectinib was also approved for the treatment of adults with metastatic non–small cell lung cancers that are ROS1-positive.
“We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue-agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” FDA Acting Commissioner Ned Sharpless, MD, said in the release.
This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than on the original tumor’s location. The previous tissue-agnostic indications approved by the FDA were pembrolizumab (Keytruda) for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib (Vitrakvi) for NTRK gene fusion tumors in 2018.
The approval of entrectinib was granted to Genentech. “Rozlytrek is the first FDA-approved treatment that selectively targets both ROS1 and NTRK fusions, and, importantly, has also shown responses in these rare cancer types that have spread to the brain,” Sandra Horning, MD, chief medical officer and head of global product development for Genentech, said in a separate press release.
Foundation Medicine will submit Foundation One CDx to the FDA for approval as a companion diagnostic for entrectinib, according to the Genentech release; an FDA-approved companion diagnostic for entrectinib is not available at this time.
“Today’s approval includes an indication for pediatric patients, 12 years of age and older, who have NTRK fusion–positive tumors by relying on efficacy information obtained primarily in adults,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Efficacy in adolescents was derived from adult data and safety was demonstrated in 30 pediatric patients.”
Entrectinib was evaluated in four clinical trials that included 54 adults with NTRK fusion–positive tumors. The overall response rate was 57%, with 7.4% of patients having complete disappearance of the tumor. Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for 9 months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid, and colon/rectum.
Clinical studies evaluated 51 adults with ROS1-positive lung cancer. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer.
The most serious side effects of entrectinib are heart failure, central nervous system effects, changes in sleep pattern, skeletal fractures, hepatotoxicity, hyperuricemia, QT prolongation, and vision disorders. Females of reproductive age and males with a female partner of reproductive potential are advised to use effective contraception during treatment; the drug may cause harm to a developing fetus or newborn baby.
Genentech must provide additional clinical trial data to the FDA as a condition of the approval.
Rozlytrek (entrectinib) has been approved to treat cancers with neurotrophic tyrosine receptor kinase (NTRK) gene fusion defects in adults and adolescents for whom there are no effective treatments, the Food and Drug Administration announced in a press release.
Entrectinib was also approved for the treatment of adults with metastatic non–small cell lung cancers that are ROS1-positive.
“We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue-agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” FDA Acting Commissioner Ned Sharpless, MD, said in the release.
This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than on the original tumor’s location. The previous tissue-agnostic indications approved by the FDA were pembrolizumab (Keytruda) for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib (Vitrakvi) for NTRK gene fusion tumors in 2018.
The approval of entrectinib was granted to Genentech. “Rozlytrek is the first FDA-approved treatment that selectively targets both ROS1 and NTRK fusions, and, importantly, has also shown responses in these rare cancer types that have spread to the brain,” Sandra Horning, MD, chief medical officer and head of global product development for Genentech, said in a separate press release.
Foundation Medicine will submit Foundation One CDx to the FDA for approval as a companion diagnostic for entrectinib, according to the Genentech release; an FDA-approved companion diagnostic for entrectinib is not available at this time.
“Today’s approval includes an indication for pediatric patients, 12 years of age and older, who have NTRK fusion–positive tumors by relying on efficacy information obtained primarily in adults,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Efficacy in adolescents was derived from adult data and safety was demonstrated in 30 pediatric patients.”
Entrectinib was evaluated in four clinical trials that included 54 adults with NTRK fusion–positive tumors. The overall response rate was 57%, with 7.4% of patients having complete disappearance of the tumor. Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for 9 months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid, and colon/rectum.
Clinical studies evaluated 51 adults with ROS1-positive lung cancer. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer.
The most serious side effects of entrectinib are heart failure, central nervous system effects, changes in sleep pattern, skeletal fractures, hepatotoxicity, hyperuricemia, QT prolongation, and vision disorders. Females of reproductive age and males with a female partner of reproductive potential are advised to use effective contraception during treatment; the drug may cause harm to a developing fetus or newborn baby.
Genentech must provide additional clinical trial data to the FDA as a condition of the approval.
Rozlytrek (entrectinib) has been approved to treat cancers with neurotrophic tyrosine receptor kinase (NTRK) gene fusion defects in adults and adolescents for whom there are no effective treatments, the Food and Drug Administration announced in a press release.
Entrectinib was also approved for the treatment of adults with metastatic non–small cell lung cancers that are ROS1-positive.
“We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue-agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine,” FDA Acting Commissioner Ned Sharpless, MD, said in the release.
This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than on the original tumor’s location. The previous tissue-agnostic indications approved by the FDA were pembrolizumab (Keytruda) for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib (Vitrakvi) for NTRK gene fusion tumors in 2018.
The approval of entrectinib was granted to Genentech. “Rozlytrek is the first FDA-approved treatment that selectively targets both ROS1 and NTRK fusions, and, importantly, has also shown responses in these rare cancer types that have spread to the brain,” Sandra Horning, MD, chief medical officer and head of global product development for Genentech, said in a separate press release.
Foundation Medicine will submit Foundation One CDx to the FDA for approval as a companion diagnostic for entrectinib, according to the Genentech release; an FDA-approved companion diagnostic for entrectinib is not available at this time.
“Today’s approval includes an indication for pediatric patients, 12 years of age and older, who have NTRK fusion–positive tumors by relying on efficacy information obtained primarily in adults,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Efficacy in adolescents was derived from adult data and safety was demonstrated in 30 pediatric patients.”
Entrectinib was evaluated in four clinical trials that included 54 adults with NTRK fusion–positive tumors. The overall response rate was 57%, with 7.4% of patients having complete disappearance of the tumor. Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for 9 months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid, and colon/rectum.
Clinical studies evaluated 51 adults with ROS1-positive lung cancer. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer.
The most serious side effects of entrectinib are heart failure, central nervous system effects, changes in sleep pattern, skeletal fractures, hepatotoxicity, hyperuricemia, QT prolongation, and vision disorders. Females of reproductive age and males with a female partner of reproductive potential are advised to use effective contraception during treatment; the drug may cause harm to a developing fetus or newborn baby.
Genentech must provide additional clinical trial data to the FDA as a condition of the approval.
FDA approves Turalio for symptomatic tenosynovial giant cell tumor
Turalio (pexidartinib) capsules have been approved for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) that is associated with severe morbidity or functional limitations not responsive to improvement with surgery, the U.S. Food and Drug Administration announced.
Turalio is the first therapy to be approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The FDA granted the approval of Turalio to Daiichi Sankyo.
“TGCT can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur, even after the procedure.”
The approval was based on results of a study of 120 patients, 59 of whom received placebo. After 25 weeks of treatment, the overall response rate was 38% (15% complete responses and 23% partial responses) in those who received pexidartinib; no responses occurred in patients who received placebo. The response persisted in 22 of 23 responders who had been followed for a minimum of 6 months, and in 13 of 13 responders who had been followed for a minimum of 12 months.
Turalio comes with a Boxed Warning about the risk of serious and potentially fatal liver injury. Liver tests should be performed prior to beginning treatment and the results monitored at specified intervals during treatment. Patients who develop abnormal results may need to withhold therapy, reduce the dose, or discontinue therapy depending on the severity of the liver injury.
Common side effects for patients were increased levels of lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and cholesterol. Loss of hair color also occurred in some patients.
Additional side effects included neutropenia, increased alkaline phosphatase levels, decreased lymphocytes, eye edema, decreased hemoglobin levels, rash, dysgeusia, and decreased phosphate levels.
Females of reproductive age and males with a female partner of reproductive potential should use effective contraception during treatment with pexidartinib. Pexidartinib may cause harm to a developing fetus or newborn baby.
Pexidartinib must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
Turalio (pexidartinib) capsules have been approved for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) that is associated with severe morbidity or functional limitations not responsive to improvement with surgery, the U.S. Food and Drug Administration announced.
Turalio is the first therapy to be approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The FDA granted the approval of Turalio to Daiichi Sankyo.
“TGCT can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur, even after the procedure.”
The approval was based on results of a study of 120 patients, 59 of whom received placebo. After 25 weeks of treatment, the overall response rate was 38% (15% complete responses and 23% partial responses) in those who received pexidartinib; no responses occurred in patients who received placebo. The response persisted in 22 of 23 responders who had been followed for a minimum of 6 months, and in 13 of 13 responders who had been followed for a minimum of 12 months.
Turalio comes with a Boxed Warning about the risk of serious and potentially fatal liver injury. Liver tests should be performed prior to beginning treatment and the results monitored at specified intervals during treatment. Patients who develop abnormal results may need to withhold therapy, reduce the dose, or discontinue therapy depending on the severity of the liver injury.
Common side effects for patients were increased levels of lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and cholesterol. Loss of hair color also occurred in some patients.
Additional side effects included neutropenia, increased alkaline phosphatase levels, decreased lymphocytes, eye edema, decreased hemoglobin levels, rash, dysgeusia, and decreased phosphate levels.
Females of reproductive age and males with a female partner of reproductive potential should use effective contraception during treatment with pexidartinib. Pexidartinib may cause harm to a developing fetus or newborn baby.
Pexidartinib must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
Turalio (pexidartinib) capsules have been approved for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) that is associated with severe morbidity or functional limitations not responsive to improvement with surgery, the U.S. Food and Drug Administration announced.
Turalio is the first therapy to be approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The FDA granted the approval of Turalio to Daiichi Sankyo.
“TGCT can cause debilitating symptoms for patients such as pain, stiffness and limitation of movement,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Surgery is the primary treatment option, but some patients are not eligible for surgery, and tumors can recur, even after the procedure.”
The approval was based on results of a study of 120 patients, 59 of whom received placebo. After 25 weeks of treatment, the overall response rate was 38% (15% complete responses and 23% partial responses) in those who received pexidartinib; no responses occurred in patients who received placebo. The response persisted in 22 of 23 responders who had been followed for a minimum of 6 months, and in 13 of 13 responders who had been followed for a minimum of 12 months.
Turalio comes with a Boxed Warning about the risk of serious and potentially fatal liver injury. Liver tests should be performed prior to beginning treatment and the results monitored at specified intervals during treatment. Patients who develop abnormal results may need to withhold therapy, reduce the dose, or discontinue therapy depending on the severity of the liver injury.
Common side effects for patients were increased levels of lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and cholesterol. Loss of hair color also occurred in some patients.
Additional side effects included neutropenia, increased alkaline phosphatase levels, decreased lymphocytes, eye edema, decreased hemoglobin levels, rash, dysgeusia, and decreased phosphate levels.
Females of reproductive age and males with a female partner of reproductive potential should use effective contraception during treatment with pexidartinib. Pexidartinib may cause harm to a developing fetus or newborn baby.
Pexidartinib must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
FDA approvals permit double-immunoassay approach to Lyme disease diagnosis
Concurrent or sequential enzyme immunoassays can now be conducted to diagnose Lyme disease, according to the U.S. Food and Drug Administration.
Four previously cleared tests are now approved by the agency for marketing with new indications as part of the revised diagnostic approach. Previously, the two-step diagnostic process consisted of an initial enzyme immunoassay followed by a Western blot test.
“With today’s action, clinicians have a new option to test for Lyme that is easier to interpret by a clinical laboratory due to the streamlined method of conducting the test. These tests may improve confidence in diagnosing a patient for a condition that requires the earliest possible treatment to ensure the best outcome for patients,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiologic Health, said in a press release announcing the newly approved approach.
The modified two-tier enzyme immunoassay approach was found to be as accurate for assessing exposure to Borrelia burgdorferi as the standard immunoassay followed by Western blot test in an FDA review of data from clinical studies using the following ZEUS Scientific ELISA Test Systems: Borrelia VlsE1/pepC10 IgG/IgM; Borrelia burgdorferi IgG/IgM; Borrelia burgdorferi IgM; and Borrelia burgdorferi IgG.
The recommendations of the Centers for Disease Control and Prevention should be followed for the diagnosis of Lyme disease and for determining when laboratory tests are appropriate, the FDA statement said. In 2017, the last year for which the CDC published data, a total of 42,743 confirmed and probable cases of Lyme disease were reported, an increase of 17% from 2016.
The FDA granted clearance of the ZEUS ELISA enzyme immunoassay tests to ZEUS Scientific.
Concurrent or sequential enzyme immunoassays can now be conducted to diagnose Lyme disease, according to the U.S. Food and Drug Administration.
Four previously cleared tests are now approved by the agency for marketing with new indications as part of the revised diagnostic approach. Previously, the two-step diagnostic process consisted of an initial enzyme immunoassay followed by a Western blot test.
“With today’s action, clinicians have a new option to test for Lyme that is easier to interpret by a clinical laboratory due to the streamlined method of conducting the test. These tests may improve confidence in diagnosing a patient for a condition that requires the earliest possible treatment to ensure the best outcome for patients,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiologic Health, said in a press release announcing the newly approved approach.
The modified two-tier enzyme immunoassay approach was found to be as accurate for assessing exposure to Borrelia burgdorferi as the standard immunoassay followed by Western blot test in an FDA review of data from clinical studies using the following ZEUS Scientific ELISA Test Systems: Borrelia VlsE1/pepC10 IgG/IgM; Borrelia burgdorferi IgG/IgM; Borrelia burgdorferi IgM; and Borrelia burgdorferi IgG.
The recommendations of the Centers for Disease Control and Prevention should be followed for the diagnosis of Lyme disease and for determining when laboratory tests are appropriate, the FDA statement said. In 2017, the last year for which the CDC published data, a total of 42,743 confirmed and probable cases of Lyme disease were reported, an increase of 17% from 2016.
The FDA granted clearance of the ZEUS ELISA enzyme immunoassay tests to ZEUS Scientific.
Concurrent or sequential enzyme immunoassays can now be conducted to diagnose Lyme disease, according to the U.S. Food and Drug Administration.
Four previously cleared tests are now approved by the agency for marketing with new indications as part of the revised diagnostic approach. Previously, the two-step diagnostic process consisted of an initial enzyme immunoassay followed by a Western blot test.
“With today’s action, clinicians have a new option to test for Lyme that is easier to interpret by a clinical laboratory due to the streamlined method of conducting the test. These tests may improve confidence in diagnosing a patient for a condition that requires the earliest possible treatment to ensure the best outcome for patients,” Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiologic Health, said in a press release announcing the newly approved approach.
The modified two-tier enzyme immunoassay approach was found to be as accurate for assessing exposure to Borrelia burgdorferi as the standard immunoassay followed by Western blot test in an FDA review of data from clinical studies using the following ZEUS Scientific ELISA Test Systems: Borrelia VlsE1/pepC10 IgG/IgM; Borrelia burgdorferi IgG/IgM; Borrelia burgdorferi IgM; and Borrelia burgdorferi IgG.
The recommendations of the Centers for Disease Control and Prevention should be followed for the diagnosis of Lyme disease and for determining when laboratory tests are appropriate, the FDA statement said. In 2017, the last year for which the CDC published data, a total of 42,743 confirmed and probable cases of Lyme disease were reported, an increase of 17% from 2016.
The FDA granted clearance of the ZEUS ELISA enzyme immunoassay tests to ZEUS Scientific.
FDA approves Xpovio for relapsed/refractory multiple myeloma
The oral therapy was approved for patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody, according to the FDA.
The approval provides a “treatment option for patients with multiple myeloma with no (other) available therapy,” said Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research.
The approval was based on a study that included 83 patients with RRMM who had an overall response rate of 25.3% to Xpovio in combination with dexamethasone.
“The median time to first response was 4 weeks, with a range of 1-10 weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma,” according to the statement.
Common side effects seen in patients taking Xpovio in combination with dexamethasone include leukopenia, neutropenia, thrombocytopenia, and anemia. Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections, and hyponatremia.
Patients taking Xpovio should be monitored for low blood counts, platelets, and sodium levels, and should avoid other medications that may cause dizziness or confusion. Patients’ hydration status, blood counts, and other medications should be optimized to avoid dizziness or confusion. Females of reproductive age and males with a female partner of reproductive potential must use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio.
Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
Xpovio in combination with dexamethasone was granted accelerated approval, and further clinical trials are required to verify and describe the drug’s clinical benefit.
The FDA granted the approval of Xpovio to Karyopharm Therapeutics.
The oral therapy was approved for patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody, according to the FDA.
The approval provides a “treatment option for patients with multiple myeloma with no (other) available therapy,” said Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research.
The approval was based on a study that included 83 patients with RRMM who had an overall response rate of 25.3% to Xpovio in combination with dexamethasone.
“The median time to first response was 4 weeks, with a range of 1-10 weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma,” according to the statement.
Common side effects seen in patients taking Xpovio in combination with dexamethasone include leukopenia, neutropenia, thrombocytopenia, and anemia. Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections, and hyponatremia.
Patients taking Xpovio should be monitored for low blood counts, platelets, and sodium levels, and should avoid other medications that may cause dizziness or confusion. Patients’ hydration status, blood counts, and other medications should be optimized to avoid dizziness or confusion. Females of reproductive age and males with a female partner of reproductive potential must use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio.
Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
Xpovio in combination with dexamethasone was granted accelerated approval, and further clinical trials are required to verify and describe the drug’s clinical benefit.
The FDA granted the approval of Xpovio to Karyopharm Therapeutics.
The oral therapy was approved for patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody, according to the FDA.
The approval provides a “treatment option for patients with multiple myeloma with no (other) available therapy,” said Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research.
The approval was based on a study that included 83 patients with RRMM who had an overall response rate of 25.3% to Xpovio in combination with dexamethasone.
“The median time to first response was 4 weeks, with a range of 1-10 weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma,” according to the statement.
Common side effects seen in patients taking Xpovio in combination with dexamethasone include leukopenia, neutropenia, thrombocytopenia, and anemia. Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections, and hyponatremia.
Patients taking Xpovio should be monitored for low blood counts, platelets, and sodium levels, and should avoid other medications that may cause dizziness or confusion. Patients’ hydration status, blood counts, and other medications should be optimized to avoid dizziness or confusion. Females of reproductive age and males with a female partner of reproductive potential must use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio.
Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.
Xpovio in combination with dexamethasone was granted accelerated approval, and further clinical trials are required to verify and describe the drug’s clinical benefit.
The FDA granted the approval of Xpovio to Karyopharm Therapeutics.
FDA approves IB-Stim device for abdominal pain in adolescents with IBS
The IB-Stim device has been approved to aid in the reduction of functional abdominal pain in patients 11-18 years of age with irritable bowel syndrome (IBS), according to the U.S. Food and Drug Administration.
“This device offers a safe option for treatment of adolescents experiencing pain from IBS through the use of mild nerve stimulation,” Carlos Peña, PhD, director of the Office of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health, said in a press release.
The prescription-only device has a single-use electrical nerve stimulator that is placed behind the patient’s ear. Stimulating nerve bundles in and around the ear is thought to provide pain relief. The battery-powered chip of the device emits low-frequency electrical pulses continuously for 5 days, at which time it is replaced. Patients can use the device for up to 3 consecutive weeks to reduce functional abdominal pain associated with IBS.
The FDA reviewed data from 50 patients, aged 11-18 years, with IBS; 27 patients were treated with the device and 23 patients received a placebo device. The study measured change from baseline to the end of the third week in worst abdominal pain, usual pain, and Pain Frequency Severity Duration (PFSD) scores. Patients were allowed to continue stable doses of medication to treat chronic abdominal pain.
IB-Stim treatment resulted in at least a 30% decrease in usual pain at the end of 3 weeks in 52% of treated patients, compared with 30% of patients who received the placebo, and at least a 30% decrease in worst pain in 59% of treated patients, compared with 26% of patients who received the placebo.
The treatment group also had greater changes in composite PFSD scores at the end of three weeks. During the study, six patients reported mild ear discomfort, and three patients reported adhesive allergy at the site of application, according to the press release.
The device is contraindicated for patients with hemophilia, patients with cardiac pacemakers, or those diagnosed with psoriasis vulgaris.
The FDA granted marketing authorization of the IB-Stim to Innovative Health Solutions.
The IB-Stim device has been approved to aid in the reduction of functional abdominal pain in patients 11-18 years of age with irritable bowel syndrome (IBS), according to the U.S. Food and Drug Administration.
“This device offers a safe option for treatment of adolescents experiencing pain from IBS through the use of mild nerve stimulation,” Carlos Peña, PhD, director of the Office of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health, said in a press release.
The prescription-only device has a single-use electrical nerve stimulator that is placed behind the patient’s ear. Stimulating nerve bundles in and around the ear is thought to provide pain relief. The battery-powered chip of the device emits low-frequency electrical pulses continuously for 5 days, at which time it is replaced. Patients can use the device for up to 3 consecutive weeks to reduce functional abdominal pain associated with IBS.
The FDA reviewed data from 50 patients, aged 11-18 years, with IBS; 27 patients were treated with the device and 23 patients received a placebo device. The study measured change from baseline to the end of the third week in worst abdominal pain, usual pain, and Pain Frequency Severity Duration (PFSD) scores. Patients were allowed to continue stable doses of medication to treat chronic abdominal pain.
IB-Stim treatment resulted in at least a 30% decrease in usual pain at the end of 3 weeks in 52% of treated patients, compared with 30% of patients who received the placebo, and at least a 30% decrease in worst pain in 59% of treated patients, compared with 26% of patients who received the placebo.
The treatment group also had greater changes in composite PFSD scores at the end of three weeks. During the study, six patients reported mild ear discomfort, and three patients reported adhesive allergy at the site of application, according to the press release.
The device is contraindicated for patients with hemophilia, patients with cardiac pacemakers, or those diagnosed with psoriasis vulgaris.
The FDA granted marketing authorization of the IB-Stim to Innovative Health Solutions.
The IB-Stim device has been approved to aid in the reduction of functional abdominal pain in patients 11-18 years of age with irritable bowel syndrome (IBS), according to the U.S. Food and Drug Administration.
“This device offers a safe option for treatment of adolescents experiencing pain from IBS through the use of mild nerve stimulation,” Carlos Peña, PhD, director of the Office of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health, said in a press release.
The prescription-only device has a single-use electrical nerve stimulator that is placed behind the patient’s ear. Stimulating nerve bundles in and around the ear is thought to provide pain relief. The battery-powered chip of the device emits low-frequency electrical pulses continuously for 5 days, at which time it is replaced. Patients can use the device for up to 3 consecutive weeks to reduce functional abdominal pain associated with IBS.
The FDA reviewed data from 50 patients, aged 11-18 years, with IBS; 27 patients were treated with the device and 23 patients received a placebo device. The study measured change from baseline to the end of the third week in worst abdominal pain, usual pain, and Pain Frequency Severity Duration (PFSD) scores. Patients were allowed to continue stable doses of medication to treat chronic abdominal pain.
IB-Stim treatment resulted in at least a 30% decrease in usual pain at the end of 3 weeks in 52% of treated patients, compared with 30% of patients who received the placebo, and at least a 30% decrease in worst pain in 59% of treated patients, compared with 26% of patients who received the placebo.
The treatment group also had greater changes in composite PFSD scores at the end of three weeks. During the study, six patients reported mild ear discomfort, and three patients reported adhesive allergy at the site of application, according to the press release.
The device is contraindicated for patients with hemophilia, patients with cardiac pacemakers, or those diagnosed with psoriasis vulgaris.
The FDA granted marketing authorization of the IB-Stim to Innovative Health Solutions.
FDA issues final guidance on seeking licensure for interchangeable biologics
The action provides “clarity for developers who want to demonstrate that their proposed biological product meets the statutory interchangeability standard under the Public Health Service Act,” Norman E. Sharpless, MD, the acting commissioner of the agency, said in a press release.
Biologics deemed “interchangeable” will be able to be substituted without the involvement of the prescriber, similar to how generic drugs are now routinely substituted for brand name drugs. On March 23, 2020, the FDA will be able to license such interchangeable products.
On May 13, the agency will consider what factors need to be weighed when determining whether an insulin product is biosimilar to or interchangeable with a reference product at a public hearing: “The Future of Insulin Biosimilars: Increasing Access and Facilitating the Efficient Development of Biosimilar and Interchangeable Insulin Products.”
“We also expect to hear stakeholder feedback on whether certain insulin products – for example, those that use insulin pumps for continuous subcutaneous infusion among the approved uses – raise unique scientific considerations that we should be considering when evaluating biosimilar or interchangeable insulin products. And importantly, we’ll also be seeking input directly from patients about their experience with insulin products, and this input will inform the FDA’s approach to implementing the regulatory pathway for biosimilar and interchangeable insulin products,” Dr. Sharpless said in the statement.
The ability to substitute an interchangeable insulin product – or any chronically used biologic medication – at the pharmacy could potentially increase access and lower costs for patients.
Dr. Sharpless added that the FDA has developed and is working to implement a Biosimilars Action Plan that includes a suite of ongoing efforts to encourage innovation and competition among biologics and the development of biosimilars.
The final guidance gives an overview of important scientific considerations in demonstrating interchangeability with a reference product and explains the scientific recommendations for an application or a supplement for a proposed interchangeable product. The guidance also explains potential ways to address the Biologics Price Competition and Innovation Act requirement for interchangeability that, for a biological product that is administered more than once to an individual, the risk in terms of safety or diminished efficacy of alternating or switching between use of the biological product and the reference product will not be greater than the risk of using the reference product without alternating or switching.
“Our rigorous scientific standards for approval will be maintained for interchangeable biologics and should serve as assurance to health care professionals and patients that they can be confident in the safety and effectiveness of both interchangeable products and biosimilar products, just as they would be for reference products,” Dr. Sharpless said in the statement.
Medical specialty organizations have begun to weigh in on the FDA final guidance, “Considerations in Demonstrating Interchangeability with a Reference Product.”
The American College of Rheumatology applauded the FDA action.
“Specifically, we are pleased to see that the final guidance expects manufacturers to use robust switching studies. At least three switches with each switch crossing over to the alternate product will be needed to determine whether alternating between a biosimilar and its reference product impacts the safety or efficacy of the drug. The ACR believes these studies will provide an understanding of what patients are likely to experience when changing formularies in a multipayer, multistate market,” Dr. Angus Worthing, chair of the American College of Rheumatology’s Government Affairs Committee, said in a statement.
“We are also pleased to see the FDA finalize its approach to safety, immunogenicity, and efficacy for the demonstration of interchangeability. And we agree with the FDA that postmarketing safety monitoring for an interchangeable product should also have robust pharmacovigilance mechanisms in place. In order to improve clarity, the ACR suggests that FDA prescribing information for all biosimilars include statements about whether each agent is or is not interchangeable to the reference product,” he said. “The ACR shares the FDA’s goal of ensuring that more affordable treatments reach patients as quickly as possible and appreciates the agency’s measured and thoughtful approach throughout this process.”
The action provides “clarity for developers who want to demonstrate that their proposed biological product meets the statutory interchangeability standard under the Public Health Service Act,” Norman E. Sharpless, MD, the acting commissioner of the agency, said in a press release.
Biologics deemed “interchangeable” will be able to be substituted without the involvement of the prescriber, similar to how generic drugs are now routinely substituted for brand name drugs. On March 23, 2020, the FDA will be able to license such interchangeable products.
On May 13, the agency will consider what factors need to be weighed when determining whether an insulin product is biosimilar to or interchangeable with a reference product at a public hearing: “The Future of Insulin Biosimilars: Increasing Access and Facilitating the Efficient Development of Biosimilar and Interchangeable Insulin Products.”
“We also expect to hear stakeholder feedback on whether certain insulin products – for example, those that use insulin pumps for continuous subcutaneous infusion among the approved uses – raise unique scientific considerations that we should be considering when evaluating biosimilar or interchangeable insulin products. And importantly, we’ll also be seeking input directly from patients about their experience with insulin products, and this input will inform the FDA’s approach to implementing the regulatory pathway for biosimilar and interchangeable insulin products,” Dr. Sharpless said in the statement.
The ability to substitute an interchangeable insulin product – or any chronically used biologic medication – at the pharmacy could potentially increase access and lower costs for patients.
Dr. Sharpless added that the FDA has developed and is working to implement a Biosimilars Action Plan that includes a suite of ongoing efforts to encourage innovation and competition among biologics and the development of biosimilars.
The final guidance gives an overview of important scientific considerations in demonstrating interchangeability with a reference product and explains the scientific recommendations for an application or a supplement for a proposed interchangeable product. The guidance also explains potential ways to address the Biologics Price Competition and Innovation Act requirement for interchangeability that, for a biological product that is administered more than once to an individual, the risk in terms of safety or diminished efficacy of alternating or switching between use of the biological product and the reference product will not be greater than the risk of using the reference product without alternating or switching.
“Our rigorous scientific standards for approval will be maintained for interchangeable biologics and should serve as assurance to health care professionals and patients that they can be confident in the safety and effectiveness of both interchangeable products and biosimilar products, just as they would be for reference products,” Dr. Sharpless said in the statement.
Medical specialty organizations have begun to weigh in on the FDA final guidance, “Considerations in Demonstrating Interchangeability with a Reference Product.”
The American College of Rheumatology applauded the FDA action.
“Specifically, we are pleased to see that the final guidance expects manufacturers to use robust switching studies. At least three switches with each switch crossing over to the alternate product will be needed to determine whether alternating between a biosimilar and its reference product impacts the safety or efficacy of the drug. The ACR believes these studies will provide an understanding of what patients are likely to experience when changing formularies in a multipayer, multistate market,” Dr. Angus Worthing, chair of the American College of Rheumatology’s Government Affairs Committee, said in a statement.
“We are also pleased to see the FDA finalize its approach to safety, immunogenicity, and efficacy for the demonstration of interchangeability. And we agree with the FDA that postmarketing safety monitoring for an interchangeable product should also have robust pharmacovigilance mechanisms in place. In order to improve clarity, the ACR suggests that FDA prescribing information for all biosimilars include statements about whether each agent is or is not interchangeable to the reference product,” he said. “The ACR shares the FDA’s goal of ensuring that more affordable treatments reach patients as quickly as possible and appreciates the agency’s measured and thoughtful approach throughout this process.”
The action provides “clarity for developers who want to demonstrate that their proposed biological product meets the statutory interchangeability standard under the Public Health Service Act,” Norman E. Sharpless, MD, the acting commissioner of the agency, said in a press release.
Biologics deemed “interchangeable” will be able to be substituted without the involvement of the prescriber, similar to how generic drugs are now routinely substituted for brand name drugs. On March 23, 2020, the FDA will be able to license such interchangeable products.
On May 13, the agency will consider what factors need to be weighed when determining whether an insulin product is biosimilar to or interchangeable with a reference product at a public hearing: “The Future of Insulin Biosimilars: Increasing Access and Facilitating the Efficient Development of Biosimilar and Interchangeable Insulin Products.”
“We also expect to hear stakeholder feedback on whether certain insulin products – for example, those that use insulin pumps for continuous subcutaneous infusion among the approved uses – raise unique scientific considerations that we should be considering when evaluating biosimilar or interchangeable insulin products. And importantly, we’ll also be seeking input directly from patients about their experience with insulin products, and this input will inform the FDA’s approach to implementing the regulatory pathway for biosimilar and interchangeable insulin products,” Dr. Sharpless said in the statement.
The ability to substitute an interchangeable insulin product – or any chronically used biologic medication – at the pharmacy could potentially increase access and lower costs for patients.
Dr. Sharpless added that the FDA has developed and is working to implement a Biosimilars Action Plan that includes a suite of ongoing efforts to encourage innovation and competition among biologics and the development of biosimilars.
The final guidance gives an overview of important scientific considerations in demonstrating interchangeability with a reference product and explains the scientific recommendations for an application or a supplement for a proposed interchangeable product. The guidance also explains potential ways to address the Biologics Price Competition and Innovation Act requirement for interchangeability that, for a biological product that is administered more than once to an individual, the risk in terms of safety or diminished efficacy of alternating or switching between use of the biological product and the reference product will not be greater than the risk of using the reference product without alternating or switching.
“Our rigorous scientific standards for approval will be maintained for interchangeable biologics and should serve as assurance to health care professionals and patients that they can be confident in the safety and effectiveness of both interchangeable products and biosimilar products, just as they would be for reference products,” Dr. Sharpless said in the statement.
Medical specialty organizations have begun to weigh in on the FDA final guidance, “Considerations in Demonstrating Interchangeability with a Reference Product.”
The American College of Rheumatology applauded the FDA action.
“Specifically, we are pleased to see that the final guidance expects manufacturers to use robust switching studies. At least three switches with each switch crossing over to the alternate product will be needed to determine whether alternating between a biosimilar and its reference product impacts the safety or efficacy of the drug. The ACR believes these studies will provide an understanding of what patients are likely to experience when changing formularies in a multipayer, multistate market,” Dr. Angus Worthing, chair of the American College of Rheumatology’s Government Affairs Committee, said in a statement.
“We are also pleased to see the FDA finalize its approach to safety, immunogenicity, and efficacy for the demonstration of interchangeability. And we agree with the FDA that postmarketing safety monitoring for an interchangeable product should also have robust pharmacovigilance mechanisms in place. In order to improve clarity, the ACR suggests that FDA prescribing information for all biosimilars include statements about whether each agent is or is not interchangeable to the reference product,” he said. “The ACR shares the FDA’s goal of ensuring that more affordable treatments reach patients as quickly as possible and appreciates the agency’s measured and thoughtful approach throughout this process.”
Ruzurgi approved for Lambert-Eaton myasthenic syndrome in patients under age 17
Amifampridine (Ruzurgi) has been approved for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune neuromuscular disorder, in patients aged 6 to less than 17 years, according to a statement from the Food and Drug Administration.
The approval is the first for a LEMS treatment specifically for pediatric patients.
“This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the statement.
The prevalence of LEMS in pediatric patients is not known, but the overall prevalence of LEMS is estimated to be three per million individuals worldwide, according to the FDA press release.
Use of amifampridine in patients 6 to less than 17 years of age is supported by pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric patients, and safety data from pediatric patients 6 to less than 17 years of age.
A randomized, double-blind, placebo-controlled withdrawal study enrolled 32 adult patients who had taken amifampridine for at least 3 months. The study compared patients continuing on amifampridine with patients switched to placebo. Effectiveness was measured by the degree of change in a test that assessed the time it took the patient to rise from a chair, walk three meters, and return to the chair for three consecutive laps without pause. The patients who continued on amifampridine experienced less impairment compared with those switched to placebo. Effectiveness was also measured with a self-assessment scale for LEMS-related weakness. The scores indicated greater perceived weakening in the patients switched to placebo.
The most common side effects among amifampridine users were paresthesia, abdominal pain, indigestion, dizziness, and nausea. Side effects reported in pediatric patients were similar to those seen in adult patients. Seizures have been observed in patients without a history of seizures. Signs of hypersensitivity reactions include rash, hives, itching, fever, swelling, or trouble breathing.
The FDA granted this application Priority Review and Fast Track designations. Amifampridine also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted the approval of amifampridine (Ruzurgi) to Jacobus Pharmaceutical Company.
Amifampridine (Ruzurgi) has been approved for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune neuromuscular disorder, in patients aged 6 to less than 17 years, according to a statement from the Food and Drug Administration.
The approval is the first for a LEMS treatment specifically for pediatric patients.
“This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the statement.
The prevalence of LEMS in pediatric patients is not known, but the overall prevalence of LEMS is estimated to be three per million individuals worldwide, according to the FDA press release.
Use of amifampridine in patients 6 to less than 17 years of age is supported by pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric patients, and safety data from pediatric patients 6 to less than 17 years of age.
A randomized, double-blind, placebo-controlled withdrawal study enrolled 32 adult patients who had taken amifampridine for at least 3 months. The study compared patients continuing on amifampridine with patients switched to placebo. Effectiveness was measured by the degree of change in a test that assessed the time it took the patient to rise from a chair, walk three meters, and return to the chair for three consecutive laps without pause. The patients who continued on amifampridine experienced less impairment compared with those switched to placebo. Effectiveness was also measured with a self-assessment scale for LEMS-related weakness. The scores indicated greater perceived weakening in the patients switched to placebo.
The most common side effects among amifampridine users were paresthesia, abdominal pain, indigestion, dizziness, and nausea. Side effects reported in pediatric patients were similar to those seen in adult patients. Seizures have been observed in patients without a history of seizures. Signs of hypersensitivity reactions include rash, hives, itching, fever, swelling, or trouble breathing.
The FDA granted this application Priority Review and Fast Track designations. Amifampridine also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted the approval of amifampridine (Ruzurgi) to Jacobus Pharmaceutical Company.
Amifampridine (Ruzurgi) has been approved for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune neuromuscular disorder, in patients aged 6 to less than 17 years, according to a statement from the Food and Drug Administration.
The approval is the first for a LEMS treatment specifically for pediatric patients.
“This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the statement.
The prevalence of LEMS in pediatric patients is not known, but the overall prevalence of LEMS is estimated to be three per million individuals worldwide, according to the FDA press release.
Use of amifampridine in patients 6 to less than 17 years of age is supported by pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric patients, and safety data from pediatric patients 6 to less than 17 years of age.
A randomized, double-blind, placebo-controlled withdrawal study enrolled 32 adult patients who had taken amifampridine for at least 3 months. The study compared patients continuing on amifampridine with patients switched to placebo. Effectiveness was measured by the degree of change in a test that assessed the time it took the patient to rise from a chair, walk three meters, and return to the chair for three consecutive laps without pause. The patients who continued on amifampridine experienced less impairment compared with those switched to placebo. Effectiveness was also measured with a self-assessment scale for LEMS-related weakness. The scores indicated greater perceived weakening in the patients switched to placebo.
The most common side effects among amifampridine users were paresthesia, abdominal pain, indigestion, dizziness, and nausea. Side effects reported in pediatric patients were similar to those seen in adult patients. Seizures have been observed in patients without a history of seizures. Signs of hypersensitivity reactions include rash, hives, itching, fever, swelling, or trouble breathing.
The FDA granted this application Priority Review and Fast Track designations. Amifampridine also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted the approval of amifampridine (Ruzurgi) to Jacobus Pharmaceutical Company.
FDA approves congenital CMV diagnostic test
, a new test to be used as an aid in the diagnosis of congenital cytomegalovirus (CMV) in newborns less than 21 days of age.
The Alethia CMV Assay Test System detects CMV DNA from a saliva swab. Results from the test should be used only in conjunction with the results of other diagnostic tests and clinical information, according to an FDA statement.
“This test for detecting the virus, when used in conjunction with the results of other diagnostic tests, may help health care providers more quickly identify the virus in newborns,” said Timothy Stenzel, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health.
In a prospective clinical study, 1,472 saliva samples out of 1,475 samples collected from newborns were correctly identified by the device as negative for the presence of CMV DNA. Three samples were incorrectly identified as positive when they were negative. Five collected saliva specimens were correctly identified as positive for the presence of CMV DNA.
In a testing of 34 samples of archived specimens from babies known to be infected with CMV, all of the archived specimens were correctly identified by the device as positive for the presence of CMV DNA.
The FDA reviewed the Alethia CMV Assay Test System through a regulatory pathway established for novel, low- to moderate-risk devices. Along with this authorization, the FDA is establishing criteria, called special controls, which determine the requirements for demonstrating accuracy, reliability, and effectiveness of tests intended to be used as an aid in the diagnosis of congenital CMV infection.
With this new regulatory classification, subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a previously approved device.
The FDA granted marketing authorization of the Alethia CMV Assay Test System to Meridian Bioscience.
, a new test to be used as an aid in the diagnosis of congenital cytomegalovirus (CMV) in newborns less than 21 days of age.
The Alethia CMV Assay Test System detects CMV DNA from a saliva swab. Results from the test should be used only in conjunction with the results of other diagnostic tests and clinical information, according to an FDA statement.
“This test for detecting the virus, when used in conjunction with the results of other diagnostic tests, may help health care providers more quickly identify the virus in newborns,” said Timothy Stenzel, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health.
In a prospective clinical study, 1,472 saliva samples out of 1,475 samples collected from newborns were correctly identified by the device as negative for the presence of CMV DNA. Three samples were incorrectly identified as positive when they were negative. Five collected saliva specimens were correctly identified as positive for the presence of CMV DNA.
In a testing of 34 samples of archived specimens from babies known to be infected with CMV, all of the archived specimens were correctly identified by the device as positive for the presence of CMV DNA.
The FDA reviewed the Alethia CMV Assay Test System through a regulatory pathway established for novel, low- to moderate-risk devices. Along with this authorization, the FDA is establishing criteria, called special controls, which determine the requirements for demonstrating accuracy, reliability, and effectiveness of tests intended to be used as an aid in the diagnosis of congenital CMV infection.
With this new regulatory classification, subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a previously approved device.
The FDA granted marketing authorization of the Alethia CMV Assay Test System to Meridian Bioscience.
, a new test to be used as an aid in the diagnosis of congenital cytomegalovirus (CMV) in newborns less than 21 days of age.
The Alethia CMV Assay Test System detects CMV DNA from a saliva swab. Results from the test should be used only in conjunction with the results of other diagnostic tests and clinical information, according to an FDA statement.
“This test for detecting the virus, when used in conjunction with the results of other diagnostic tests, may help health care providers more quickly identify the virus in newborns,” said Timothy Stenzel, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health.
In a prospective clinical study, 1,472 saliva samples out of 1,475 samples collected from newborns were correctly identified by the device as negative for the presence of CMV DNA. Three samples were incorrectly identified as positive when they were negative. Five collected saliva specimens were correctly identified as positive for the presence of CMV DNA.
In a testing of 34 samples of archived specimens from babies known to be infected with CMV, all of the archived specimens were correctly identified by the device as positive for the presence of CMV DNA.
The FDA reviewed the Alethia CMV Assay Test System through a regulatory pathway established for novel, low- to moderate-risk devices. Along with this authorization, the FDA is establishing criteria, called special controls, which determine the requirements for demonstrating accuracy, reliability, and effectiveness of tests intended to be used as an aid in the diagnosis of congenital CMV infection.
With this new regulatory classification, subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) process, whereby devices can obtain marketing authorization by demonstrating substantial equivalence to a previously approved device.
The FDA granted marketing authorization of the Alethia CMV Assay Test System to Meridian Bioscience.
Reducing asthma, COPD exacerbations in obese patients
SAN ANTONIO – Interventions that address variations in inflammation type and metabolism unique to might prove useful for improving their management, Cherry Wongtrakool, MD, of Emory University, Atlanta, said in a presentation at the annual meeting of the American College of Chest Physicians.
Obese patients with asthma or COPD typically have metabolic and inflammatory profiles that differ from those of nonobese patients with the disorders. Obesity is associated with the development of asthma as well as its severity and the risk for exacerbations. Obese patients with asthma are less likely to have controlled disease or to respond to medication.
The variations in asthma related to obesity even can be traced to infancy for some. Children with rapid weight gain after birth, for example, have an increased risk for developing asthma. In the recently published Boston Birth Cohort study, more than 500 babies from urban, low income families were followed from birth until age 16. Babies with rapid weight gain at 4 months and at 24 months had an increased risk for developing asthma by age 16. Even after adjusting for multiple risk factors, the increased risk for developing asthma persisted in these obese infants.
Higher BMIs during infancy may affect lung development, which continues up to age 5-8 years, Dr. Wongtrakool said. Obesity may affect immune system development. Asthma may develop when persistent inflammation during infancy gets a second hit from genetic factors or from risk factors such as atopy or maternal smoking.
Dr. Wongtrakool noted that obese patients with asthma, unlike nonobese asthma patients, tend to have non-TH2 inflammation. Their TH1/TH2 ratio in stimulated T cells is higher and is directly associated with insulin resistance. Similar to obese patients without asthma, they have higher levels of circulating TNF-alpha, interferon-gamma inducible protein 10, and monocyte chemoattractant protein-1 (MCP-1). They are more likely to have insulin resistance, low high-density lipid levels, differences in gut microbiota, increased leptin, decreased adiponectin, increased asymmetric dimethylarginine, and decreased exhaled nitrous oxide (NO).
In broncheoalveolar lavage samples, obese asthma patients have more cells that secrete interleukin-17, Dr. Wontrakool said. TH17-associated inflammation also has an influence in asthma with obesity. A recent study of 30 obese and lean asthma patients found a difference in metabolites measured in breath samples of obese people with asthma, compared with lean people with asthma and obese people without asthma.
In terms of metabolites in their breath, obese asthma patients clustered together and differed from lean patients with asthma and obese patients without asthma.
Obese people with asthma also differ in their gut microbiota, having more firmicutes species and decreased bacteroides species. Studies in mice indicate that these species have a role in body weight and that altering gut microbiota via fecal transplant was associated with weight loss when obese mice received fecal transplants from lean mice, and vice versa.
In the Supplemental Nutrition in Asthma Control (SNAC) study, preadolescents with asthma were given a nutrition bar designed by researchers at the Children’s Hospital Oakland (Calif.) Research Institute. The children also received asthma education and exercise classes, but the intervention was not designed to reduce weight. FVC and FEV1 improved in all study participants, but those participants in the low inflammation subgroup had the most pronounced improvements in FVC and FEV1 after 2 months.
Dr. Wongtrakool called the study “intriguing,” as it indicates asthma patients with lower level inflammation appear more likely to benefit from nutritional supplementation.
In another study of 55 obese adult asthma patients, a hypocaloric diet, access to a nutritionist and psychologist, and exercise classes were associated with improved asthma control and an improved inflammatory and metabolic profile.
In a British registry of the outcomes of bariatric surgery for obesity, patients who also had asthma had a decrease in asthma prevalence in the year after surgery that persisted over 5 years.
The association of COPD with obesity has been less studied than asthma and COPD, but metabolic syndrome appears to be on the rise in these patients. In a study performed over a decade ago, 47% of COPD patients met the definition of metabolic syndrome; a more recent study found 77% of COPD patients met the standard.
Admission glucose levels also have been found to influence the severity of COPD exacerbation. With higher blood glucose levels, there was a higher risk of mortality—from 12% in those with glucose levels of less than 6.0 mmol/l to 31% among those with glucose levels exceeding 9.0 mmol/l, one study showed.
Bariatric surgery may reduce the risk of acute exacerbations of COPD in obese patients, another recent study found. In a study of 480 obese patients with COPD who underwent bariatric surgery, their 28% presurgical risk of acute exacerbations of COPD was cut in half by 12 months after surgery, and the reduction persisted at 24 months.
SAN ANTONIO – Interventions that address variations in inflammation type and metabolism unique to might prove useful for improving their management, Cherry Wongtrakool, MD, of Emory University, Atlanta, said in a presentation at the annual meeting of the American College of Chest Physicians.
Obese patients with asthma or COPD typically have metabolic and inflammatory profiles that differ from those of nonobese patients with the disorders. Obesity is associated with the development of asthma as well as its severity and the risk for exacerbations. Obese patients with asthma are less likely to have controlled disease or to respond to medication.
The variations in asthma related to obesity even can be traced to infancy for some. Children with rapid weight gain after birth, for example, have an increased risk for developing asthma. In the recently published Boston Birth Cohort study, more than 500 babies from urban, low income families were followed from birth until age 16. Babies with rapid weight gain at 4 months and at 24 months had an increased risk for developing asthma by age 16. Even after adjusting for multiple risk factors, the increased risk for developing asthma persisted in these obese infants.
Higher BMIs during infancy may affect lung development, which continues up to age 5-8 years, Dr. Wongtrakool said. Obesity may affect immune system development. Asthma may develop when persistent inflammation during infancy gets a second hit from genetic factors or from risk factors such as atopy or maternal smoking.
Dr. Wongtrakool noted that obese patients with asthma, unlike nonobese asthma patients, tend to have non-TH2 inflammation. Their TH1/TH2 ratio in stimulated T cells is higher and is directly associated with insulin resistance. Similar to obese patients without asthma, they have higher levels of circulating TNF-alpha, interferon-gamma inducible protein 10, and monocyte chemoattractant protein-1 (MCP-1). They are more likely to have insulin resistance, low high-density lipid levels, differences in gut microbiota, increased leptin, decreased adiponectin, increased asymmetric dimethylarginine, and decreased exhaled nitrous oxide (NO).
In broncheoalveolar lavage samples, obese asthma patients have more cells that secrete interleukin-17, Dr. Wontrakool said. TH17-associated inflammation also has an influence in asthma with obesity. A recent study of 30 obese and lean asthma patients found a difference in metabolites measured in breath samples of obese people with asthma, compared with lean people with asthma and obese people without asthma.
In terms of metabolites in their breath, obese asthma patients clustered together and differed from lean patients with asthma and obese patients without asthma.
Obese people with asthma also differ in their gut microbiota, having more firmicutes species and decreased bacteroides species. Studies in mice indicate that these species have a role in body weight and that altering gut microbiota via fecal transplant was associated with weight loss when obese mice received fecal transplants from lean mice, and vice versa.
In the Supplemental Nutrition in Asthma Control (SNAC) study, preadolescents with asthma were given a nutrition bar designed by researchers at the Children’s Hospital Oakland (Calif.) Research Institute. The children also received asthma education and exercise classes, but the intervention was not designed to reduce weight. FVC and FEV1 improved in all study participants, but those participants in the low inflammation subgroup had the most pronounced improvements in FVC and FEV1 after 2 months.
Dr. Wongtrakool called the study “intriguing,” as it indicates asthma patients with lower level inflammation appear more likely to benefit from nutritional supplementation.
In another study of 55 obese adult asthma patients, a hypocaloric diet, access to a nutritionist and psychologist, and exercise classes were associated with improved asthma control and an improved inflammatory and metabolic profile.
In a British registry of the outcomes of bariatric surgery for obesity, patients who also had asthma had a decrease in asthma prevalence in the year after surgery that persisted over 5 years.
The association of COPD with obesity has been less studied than asthma and COPD, but metabolic syndrome appears to be on the rise in these patients. In a study performed over a decade ago, 47% of COPD patients met the definition of metabolic syndrome; a more recent study found 77% of COPD patients met the standard.
Admission glucose levels also have been found to influence the severity of COPD exacerbation. With higher blood glucose levels, there was a higher risk of mortality—from 12% in those with glucose levels of less than 6.0 mmol/l to 31% among those with glucose levels exceeding 9.0 mmol/l, one study showed.
Bariatric surgery may reduce the risk of acute exacerbations of COPD in obese patients, another recent study found. In a study of 480 obese patients with COPD who underwent bariatric surgery, their 28% presurgical risk of acute exacerbations of COPD was cut in half by 12 months after surgery, and the reduction persisted at 24 months.
SAN ANTONIO – Interventions that address variations in inflammation type and metabolism unique to might prove useful for improving their management, Cherry Wongtrakool, MD, of Emory University, Atlanta, said in a presentation at the annual meeting of the American College of Chest Physicians.
Obese patients with asthma or COPD typically have metabolic and inflammatory profiles that differ from those of nonobese patients with the disorders. Obesity is associated with the development of asthma as well as its severity and the risk for exacerbations. Obese patients with asthma are less likely to have controlled disease or to respond to medication.
The variations in asthma related to obesity even can be traced to infancy for some. Children with rapid weight gain after birth, for example, have an increased risk for developing asthma. In the recently published Boston Birth Cohort study, more than 500 babies from urban, low income families were followed from birth until age 16. Babies with rapid weight gain at 4 months and at 24 months had an increased risk for developing asthma by age 16. Even after adjusting for multiple risk factors, the increased risk for developing asthma persisted in these obese infants.
Higher BMIs during infancy may affect lung development, which continues up to age 5-8 years, Dr. Wongtrakool said. Obesity may affect immune system development. Asthma may develop when persistent inflammation during infancy gets a second hit from genetic factors or from risk factors such as atopy or maternal smoking.
Dr. Wongtrakool noted that obese patients with asthma, unlike nonobese asthma patients, tend to have non-TH2 inflammation. Their TH1/TH2 ratio in stimulated T cells is higher and is directly associated with insulin resistance. Similar to obese patients without asthma, they have higher levels of circulating TNF-alpha, interferon-gamma inducible protein 10, and monocyte chemoattractant protein-1 (MCP-1). They are more likely to have insulin resistance, low high-density lipid levels, differences in gut microbiota, increased leptin, decreased adiponectin, increased asymmetric dimethylarginine, and decreased exhaled nitrous oxide (NO).
In broncheoalveolar lavage samples, obese asthma patients have more cells that secrete interleukin-17, Dr. Wontrakool said. TH17-associated inflammation also has an influence in asthma with obesity. A recent study of 30 obese and lean asthma patients found a difference in metabolites measured in breath samples of obese people with asthma, compared with lean people with asthma and obese people without asthma.
In terms of metabolites in their breath, obese asthma patients clustered together and differed from lean patients with asthma and obese patients without asthma.
Obese people with asthma also differ in their gut microbiota, having more firmicutes species and decreased bacteroides species. Studies in mice indicate that these species have a role in body weight and that altering gut microbiota via fecal transplant was associated with weight loss when obese mice received fecal transplants from lean mice, and vice versa.
In the Supplemental Nutrition in Asthma Control (SNAC) study, preadolescents with asthma were given a nutrition bar designed by researchers at the Children’s Hospital Oakland (Calif.) Research Institute. The children also received asthma education and exercise classes, but the intervention was not designed to reduce weight. FVC and FEV1 improved in all study participants, but those participants in the low inflammation subgroup had the most pronounced improvements in FVC and FEV1 after 2 months.
Dr. Wongtrakool called the study “intriguing,” as it indicates asthma patients with lower level inflammation appear more likely to benefit from nutritional supplementation.
In another study of 55 obese adult asthma patients, a hypocaloric diet, access to a nutritionist and psychologist, and exercise classes were associated with improved asthma control and an improved inflammatory and metabolic profile.
In a British registry of the outcomes of bariatric surgery for obesity, patients who also had asthma had a decrease in asthma prevalence in the year after surgery that persisted over 5 years.
The association of COPD with obesity has been less studied than asthma and COPD, but metabolic syndrome appears to be on the rise in these patients. In a study performed over a decade ago, 47% of COPD patients met the definition of metabolic syndrome; a more recent study found 77% of COPD patients met the standard.
Admission glucose levels also have been found to influence the severity of COPD exacerbation. With higher blood glucose levels, there was a higher risk of mortality—from 12% in those with glucose levels of less than 6.0 mmol/l to 31% among those with glucose levels exceeding 9.0 mmol/l, one study showed.
Bariatric surgery may reduce the risk of acute exacerbations of COPD in obese patients, another recent study found. In a study of 480 obese patients with COPD who underwent bariatric surgery, their 28% presurgical risk of acute exacerbations of COPD was cut in half by 12 months after surgery, and the reduction persisted at 24 months.
REPORTING FROM CHEST 2018