FDA/CDC

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved Suflave, a new low-volume, lemon-lime flavored liquid osmotic laxative for colonoscopy preparation in adults, the manufacturer, Sebela Pharmaceuticals, has announced.

Suflave comes in a carton containing two bottles and two flavor packets. Each bottle contains 178.7 g polyethylene glycol 3350, 7.3 g sodium sulfate, 1.12 g potassium chloride, 0.9 g magnesium sulfate, and 0.5 g sodium chloride. One bottle and one flavor packet are equivalent to one dose.

Administration of both doses is required for a complete preparation for colonoscopy. After each dose, an additional 16 ounces of water must be consumed. 

In a clinical trial, 94% of patients achieved successful bowel cleansing with Suflave, the company said in a news release.

Most patients reported that Suflave tastes like a sports drink and described the taste as “neutral to very pleasant.” Most patients also reported that Suflave was “tolerable to very easy” to consume and indicated they would request it for a subsequent colonoscopy.

“Patients frequently struggle with the taste and volume of traditional bowel preparations – and fear related to the preparation can also negatively affect patient willingness to undergo follow-up colonoscopy if it is indicated,” Douglas K. Rex, MD, gastroenterologist and distinguished professor emeritus at Indiana University, Indianapolis, said in the release.

“I believe the palatable lemon-lime flavor of Suflave will be a welcomed option for patients – reducing preparation hesitancy and giving more people the chance to feel comfortable during preparation and getting a successful and effective procedure,” Dr. Rex added.

Suflave will be available by prescription to patients in the United States in early August.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved Suflave, a new low-volume, lemon-lime flavored liquid osmotic laxative for colonoscopy preparation in adults, the manufacturer, Sebela Pharmaceuticals, has announced.

Suflave comes in a carton containing two bottles and two flavor packets. Each bottle contains 178.7 g polyethylene glycol 3350, 7.3 g sodium sulfate, 1.12 g potassium chloride, 0.9 g magnesium sulfate, and 0.5 g sodium chloride. One bottle and one flavor packet are equivalent to one dose.

Administration of both doses is required for a complete preparation for colonoscopy. After each dose, an additional 16 ounces of water must be consumed. 

In a clinical trial, 94% of patients achieved successful bowel cleansing with Suflave, the company said in a news release.

Most patients reported that Suflave tastes like a sports drink and described the taste as “neutral to very pleasant.” Most patients also reported that Suflave was “tolerable to very easy” to consume and indicated they would request it for a subsequent colonoscopy.

“Patients frequently struggle with the taste and volume of traditional bowel preparations – and fear related to the preparation can also negatively affect patient willingness to undergo follow-up colonoscopy if it is indicated,” Douglas K. Rex, MD, gastroenterologist and distinguished professor emeritus at Indiana University, Indianapolis, said in the release.

“I believe the palatable lemon-lime flavor of Suflave will be a welcomed option for patients – reducing preparation hesitancy and giving more people the chance to feel comfortable during preparation and getting a successful and effective procedure,” Dr. Rex added.

Suflave will be available by prescription to patients in the United States in early August.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved Suflave, a new low-volume, lemon-lime flavored liquid osmotic laxative for colonoscopy preparation in adults, the manufacturer, Sebela Pharmaceuticals, has announced.

Suflave comes in a carton containing two bottles and two flavor packets. Each bottle contains 178.7 g polyethylene glycol 3350, 7.3 g sodium sulfate, 1.12 g potassium chloride, 0.9 g magnesium sulfate, and 0.5 g sodium chloride. One bottle and one flavor packet are equivalent to one dose.

Administration of both doses is required for a complete preparation for colonoscopy. After each dose, an additional 16 ounces of water must be consumed. 

In a clinical trial, 94% of patients achieved successful bowel cleansing with Suflave, the company said in a news release.

Most patients reported that Suflave tastes like a sports drink and described the taste as “neutral to very pleasant.” Most patients also reported that Suflave was “tolerable to very easy” to consume and indicated they would request it for a subsequent colonoscopy.

“Patients frequently struggle with the taste and volume of traditional bowel preparations – and fear related to the preparation can also negatively affect patient willingness to undergo follow-up colonoscopy if it is indicated,” Douglas K. Rex, MD, gastroenterologist and distinguished professor emeritus at Indiana University, Indianapolis, said in the release.

“I believe the palatable lemon-lime flavor of Suflave will be a welcomed option for patients – reducing preparation hesitancy and giving more people the chance to feel comfortable during preparation and getting a successful and effective procedure,” Dr. Rex added.

Suflave will be available by prescription to patients in the United States in early August.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved donislecel (Lantidra, CellTrans), a pancreatic islet cell therapy developed from cadaver donors, for the treatment of people with type 1 diabetes who are unable to achieve target glucose levels owing to severe hypoglycemic episodes.

The product is given as a single infusion via the hepatic portal vein into the liver. A second infusion is given if necessary. Immunosuppression is required to maintain cell viability, just as it is required to support a transplanted kidney or other organ, as these all represent “foreign” tissues to the recipient.

“Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in an FDA statement.

The product was approved despite concerns from the American Society of Transplant Surgeons, the American Society of Transplantation, and an organization of more than 50 transplant surgeons – the Islets for U.S. Collaborative – whose members argue that cadaver-derived (allogeneic) pancreatic islets should be regulated as transplanted organs rather than as biologic drugs, as is done in many other parts of the world.

Lantidra differs from stem cell therapy being developed by Vertex Pharmaceuticals. In the latter, beta cells are grown from allogeneic stem cells using a proprietary technology. So far, six patients have received the therapy, and it has been successful in all of them to varying degrees, as reported at last week’s American Diabetes Association meeting. So while this is a promising technology, with talk of a “cure” for type 1 diabetes, it’s important to remember that this is very early in the development phase, says Anne Peters, MD, of the University of California, Los Angeles.
 

Approval based on small studies, with adverse events

The approval of Lantidra, following a 12-4 vote in favor by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in April 2021, was based on two nonrandomized, single-arm studies that included a total of 30 individuals with type 1 diabetes who had hypoglycemic unawareness and who received between one and three infusions of donislecel.

Insulin independence was achieved at 1 year by 21 participants; 11 were still insulin independent at 5 years, and 10 remained so more than 5 years. Five participants were unable to discontinue insulin treatment at all.

Adverse events included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most of the participants experienced at least one serious adverse reaction related to the method of infusion and/or the use of immunosuppression. Some of these reactions required discontinuation of the immunosuppressive medications, resulting in the loss of islet cell function and return to insulin dependence.

“These adverse events should be considered when assessing the benefits and risks of Lantidra for each patient. Lantidra is approved with patient-directed labeling to inform patients with type 1 diabetes about benefits and risks of Lantidra,” according to the FDA statement.
 

U.S. transplant physicians had expressed concern, bill introduced

The transplant surgery organizations had written letters to the FDA, as well as to several other government agencies, to ask that the regulatory framework for Lantidra be shifted from the FDA to the Organ Procurement and Transplantation Network and the United Network for Organ Sharing.

They also wrote to members of Congress. On June 22, 2023, U.S. Senators Mike Lee (R-UT), Ted Budd (R-NC), and Marsha Blackburn (R-TN) introduced the Islet Transplantation Bill, which would shift the regulatory framework for cadaveric islets from that of biologic drugs to transplanted organs.

Asked for comment, Piotr Witkowski, MD, PhD, the leader of the Islets for U.S. Collaborative, told this news organization: “We were really happy about the introduction of the islet bill. Now, we’re concerned about negative downstream effects of granting a licence to a private company for distribution of the cadaveric islets.”

During the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee’s discussion in 2021, several panel members noted that the target patient population for this treatment with the current indication will likely be smaller today than it was when the two studies were initiated, in 2004 and 2007, given current automated diabetes technology – such as insulin pumps, continuous glucose monitors, and hybrid closed-loop systems in which the two are linked together as a so-called artificial pancreas – that reduces hypoglycemia risk.

A version of this article originally appeared on Medscape.com.

The U.S. Food and Drug Administration has approved rozanolixizumab (Rystiggo) to treat adults with generalized myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti–muscle-specific tyrosine kinase (MuSK) antibody, the drug’s manufacturer, UCB, has announced.

Olivier Le Moal/Getty Images

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved rozanolixizumab (Rystiggo) to treat adults with generalized myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti–muscle-specific tyrosine kinase (MuSK) antibody, the drug’s manufacturer, UCB, has announced.

Olivier Le Moal/Getty Images

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved rozanolixizumab (Rystiggo) to treat adults with generalized myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti–muscle-specific tyrosine kinase (MuSK) antibody, the drug’s manufacturer, UCB, has announced.

Olivier Le Moal/Getty Images

A version of this article first appeared on Medscape.com.

Roche has received Food and Drug Administration 510(k) clearance for additional cerebrospinal fluid (CSF) assays for Alzheimer’s disease (AD), supporting timely diagnosis and treatment decision-making.

The Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys total-tau CSF assays (tTau) (used as a tTau/Abeta42 ratio) are for use in adults ages 55 and older being evaluated for AD.

They join the Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys phospho-tau (181P) CSF (pTau181) assays (used as a pTau181/Abeta42 ratio) that received FDA 510(k) clearance in 2022.

Olivier Le Moal/Getty Images

“An early and accurate diagnosis can help patients, caregivers and physicians determine a path forward, and the Elecsys CSF assays support diagnosis at early disease stages, when treatment is most effective,” Brad Moore, president and CEO of Roche Diagnostics North America, said in a statement.

Appropriate use recommendations for new and emerging AD drugs call for confirmation of amyloid pathology. Currently, the only FDA-cleared methods to confirm amyloid pathology are CSF tests and PET scans.

“The Elecsys AD CSF assays are concordant with amyloid PET scan imaging and have the potential to provide a more affordable and accessible routine option to confirm the presence of amyloid pathology in the brain,” Roche said.

“They also offer detection of both amyloid and tau biomarkers from one draw, with no radiation and potential to detect Alzheimer’s pathology in early stages of disease,” the company added.

The previously approved Elecsys pTau181/Abeta42 ratio is currently available and the newly approved Elecsys tTau/Abeta42 ratio will be available in the fourth quarter of 2023.

A version of this article first appeared on Medscape.com.

Roche has received Food and Drug Administration 510(k) clearance for additional cerebrospinal fluid (CSF) assays for Alzheimer’s disease (AD), supporting timely diagnosis and treatment decision-making.

The Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys total-tau CSF assays (tTau) (used as a tTau/Abeta42 ratio) are for use in adults ages 55 and older being evaluated for AD.

They join the Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys phospho-tau (181P) CSF (pTau181) assays (used as a pTau181/Abeta42 ratio) that received FDA 510(k) clearance in 2022.

Olivier Le Moal/Getty Images

“An early and accurate diagnosis can help patients, caregivers and physicians determine a path forward, and the Elecsys CSF assays support diagnosis at early disease stages, when treatment is most effective,” Brad Moore, president and CEO of Roche Diagnostics North America, said in a statement.

Appropriate use recommendations for new and emerging AD drugs call for confirmation of amyloid pathology. Currently, the only FDA-cleared methods to confirm amyloid pathology are CSF tests and PET scans.

“The Elecsys AD CSF assays are concordant with amyloid PET scan imaging and have the potential to provide a more affordable and accessible routine option to confirm the presence of amyloid pathology in the brain,” Roche said.

“They also offer detection of both amyloid and tau biomarkers from one draw, with no radiation and potential to detect Alzheimer’s pathology in early stages of disease,” the company added.

The previously approved Elecsys pTau181/Abeta42 ratio is currently available and the newly approved Elecsys tTau/Abeta42 ratio will be available in the fourth quarter of 2023.

A version of this article first appeared on Medscape.com.

Roche has received Food and Drug Administration 510(k) clearance for additional cerebrospinal fluid (CSF) assays for Alzheimer’s disease (AD), supporting timely diagnosis and treatment decision-making.

The Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys total-tau CSF assays (tTau) (used as a tTau/Abeta42 ratio) are for use in adults ages 55 and older being evaluated for AD.

They join the Elecsys beta-amyloid (1-42) CSF II (Abeta42) and Elecsys phospho-tau (181P) CSF (pTau181) assays (used as a pTau181/Abeta42 ratio) that received FDA 510(k) clearance in 2022.

Olivier Le Moal/Getty Images

“An early and accurate diagnosis can help patients, caregivers and physicians determine a path forward, and the Elecsys CSF assays support diagnosis at early disease stages, when treatment is most effective,” Brad Moore, president and CEO of Roche Diagnostics North America, said in a statement.

Appropriate use recommendations for new and emerging AD drugs call for confirmation of amyloid pathology. Currently, the only FDA-cleared methods to confirm amyloid pathology are CSF tests and PET scans.

“The Elecsys AD CSF assays are concordant with amyloid PET scan imaging and have the potential to provide a more affordable and accessible routine option to confirm the presence of amyloid pathology in the brain,” Roche said.

“They also offer detection of both amyloid and tau biomarkers from one draw, with no radiation and potential to detect Alzheimer’s pathology in early stages of disease,” the company added.

The previously approved Elecsys pTau181/Abeta42 ratio is currently available and the newly approved Elecsys tTau/Abeta42 ratio will be available in the fourth quarter of 2023.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved the oral poly (ADP-ribose) polymerase inhibitor talazoparib (Talzenna, Pfizer) plus enzalutamide (Xtandi) to treat homologous recombination repair (HRR) gene–mutated metastatic castration-resistant prostate cancer.

Talazoparib is already approved for adults with deleterious or suspected deleterious germline BRCA-mutated HER2-negative locally advanced or metastatic breast cancer. The new approval, granted following priority review, is based on findings from the randomized, placebo-controlled, phase 3 TALAPRO-2 trial, published in The Lancet.

The 399 patients in the study were randomly assigned in a 1:1 ratio to receive either enzalutamide 160 mg daily plus either talazoparib 0.5 mg or placebo daily. Median radiographic progression-free survival (PFS) was not reached in the treatment group; it was 13.8 months in the placebo group (hazard ratio, 0.45). In an exploratory analysis by BRCA mutation status, patients with BRCA-mutated disease who received talazoparib exhibited an even stronger median radiographic PFS (HR, 0.20; not reached vs. 11 months) in comparison with those without BRCA-mutated disease (HR, 0.72; 24.7 vs. 16.7 months).

Serious adverse reactions occurred in 30% of patients who received talazoparib plus enzalutamide. The most common serious adverse reactions, reported in more than 2% of patients, included anemia (9%) and fracture (3%). Discontinuation of talazoparib occurred in 10% of patients, according to a Pfizer statement.

Pfizer also noted that a marketing authorization application for the drug combination has been accepted for review by the European Medicines Agency.

“Despite treatment advancement in metastatic castration-resistant prostate cancer, the disease can progress quickly, and many patients may only receive one line of therapy,” lead investigator Neeraj Agarwal, MD, of the Huntsman Cancer Institute, University of Utah, Salt Lake City, said in a statement. Patients with metastatic castration-resistant prostate cancer harboring HRR genetic alterations have even worse outcomes, and thus the FDA’s approval of the talazoparib and enzalutamide combination “represents a treatment option deserving of excitement and attention.”

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved the oral poly (ADP-ribose) polymerase inhibitor talazoparib (Talzenna, Pfizer) plus enzalutamide (Xtandi) to treat homologous recombination repair (HRR) gene–mutated metastatic castration-resistant prostate cancer.

Talazoparib is already approved for adults with deleterious or suspected deleterious germline BRCA-mutated HER2-negative locally advanced or metastatic breast cancer. The new approval, granted following priority review, is based on findings from the randomized, placebo-controlled, phase 3 TALAPRO-2 trial, published in The Lancet.

The 399 patients in the study were randomly assigned in a 1:1 ratio to receive either enzalutamide 160 mg daily plus either talazoparib 0.5 mg or placebo daily. Median radiographic progression-free survival (PFS) was not reached in the treatment group; it was 13.8 months in the placebo group (hazard ratio, 0.45). In an exploratory analysis by BRCA mutation status, patients with BRCA-mutated disease who received talazoparib exhibited an even stronger median radiographic PFS (HR, 0.20; not reached vs. 11 months) in comparison with those without BRCA-mutated disease (HR, 0.72; 24.7 vs. 16.7 months).

Serious adverse reactions occurred in 30% of patients who received talazoparib plus enzalutamide. The most common serious adverse reactions, reported in more than 2% of patients, included anemia (9%) and fracture (3%). Discontinuation of talazoparib occurred in 10% of patients, according to a Pfizer statement.

Pfizer also noted that a marketing authorization application for the drug combination has been accepted for review by the European Medicines Agency.

“Despite treatment advancement in metastatic castration-resistant prostate cancer, the disease can progress quickly, and many patients may only receive one line of therapy,” lead investigator Neeraj Agarwal, MD, of the Huntsman Cancer Institute, University of Utah, Salt Lake City, said in a statement. Patients with metastatic castration-resistant prostate cancer harboring HRR genetic alterations have even worse outcomes, and thus the FDA’s approval of the talazoparib and enzalutamide combination “represents a treatment option deserving of excitement and attention.”

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved the oral poly (ADP-ribose) polymerase inhibitor talazoparib (Talzenna, Pfizer) plus enzalutamide (Xtandi) to treat homologous recombination repair (HRR) gene–mutated metastatic castration-resistant prostate cancer.

Talazoparib is already approved for adults with deleterious or suspected deleterious germline BRCA-mutated HER2-negative locally advanced or metastatic breast cancer. The new approval, granted following priority review, is based on findings from the randomized, placebo-controlled, phase 3 TALAPRO-2 trial, published in The Lancet.

The 399 patients in the study were randomly assigned in a 1:1 ratio to receive either enzalutamide 160 mg daily plus either talazoparib 0.5 mg or placebo daily. Median radiographic progression-free survival (PFS) was not reached in the treatment group; it was 13.8 months in the placebo group (hazard ratio, 0.45). In an exploratory analysis by BRCA mutation status, patients with BRCA-mutated disease who received talazoparib exhibited an even stronger median radiographic PFS (HR, 0.20; not reached vs. 11 months) in comparison with those without BRCA-mutated disease (HR, 0.72; 24.7 vs. 16.7 months).

Serious adverse reactions occurred in 30% of patients who received talazoparib plus enzalutamide. The most common serious adverse reactions, reported in more than 2% of patients, included anemia (9%) and fracture (3%). Discontinuation of talazoparib occurred in 10% of patients, according to a Pfizer statement.

Pfizer also noted that a marketing authorization application for the drug combination has been accepted for review by the European Medicines Agency.

“Despite treatment advancement in metastatic castration-resistant prostate cancer, the disease can progress quickly, and many patients may only receive one line of therapy,” lead investigator Neeraj Agarwal, MD, of the Huntsman Cancer Institute, University of Utah, Salt Lake City, said in a statement. Patients with metastatic castration-resistant prostate cancer harboring HRR genetic alterations have even worse outcomes, and thus the FDA’s approval of the talazoparib and enzalutamide combination “represents a treatment option deserving of excitement and attention.”

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has granted accelerated approval for glofitamab (Columvi) for use in certain types of lymphoma.

The indication is for use in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified or with LBCL arising from follicular lymphoma who have received two or more lines of systemic therapy.

The product is a T cell–engaging bispecific antibody developed by Genentech, which has a similar product, mosunetuzumab-axgb (Lunsumio), for the treatment of follicular lymphoma. Lunsumio was approved in December 2022.

These drugs could be considered a first choice in the setting of third-line therapy, suggests an expert writing recently in The New England Journal of Medicine.

Nancy Bartlett, MD, from the Siteman Cancer Center, Washington University in St. Louis, is the author of an editorial that accompanied the publication of results with glofitamab in the pivotal trial that led to its approval.

“Bispecific agents will be an excellent option for the 60% of patients in whom second-line CAR [chimeric antigen receptor] T-cell therapy fails,” she wrote in her editorial.

Dr. Bartlett suggests that these agents may be preferred over CAR T cells. “If longer follow-up confirms that the majority of complete remissions with bispecific agents are durable, on the basis of the advantages of availability (including in the community setting) and more favorable immediate and late toxic-effect profiles, bispecific agents could be considered as the initial choice. ... CAR T-cell therapy could be held in reserve for patients who do not have a complete response or who have a relapse after a complete response.”
 

Most common form of non-Hodgkin’s lymphoma

DLBCL is the most common form of non-Hodgkin’s lymphoma in the United States, the company noted in a press release. While many people with DLBCL are responsive to treatment, the majority of those who experience relapse or whose condition is refractory to subsequent treatments have poor outcomes.

“Patients with relapsed or refractory diffuse large B-cell lymphoma may experience rapid progression of their cancer and often urgently need an effective treatment option that can be administered without delay,” commented Krish Patel, MD, director of the lymphoma program at the Swedish Cancer Institute in Seattle, who is an investigator on the clinical trial that led to the product’s approval. He said that the results from trials suggest that glofitamab gives patients “a chance for complete remission with a fixed-duration immunotherapy and that such remissions can potentially be sustained after the end of their treatment.”

The accelerated approval is based on response rate and durability of response results from the phase 1/2 NP30179 study.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

This trial involved 132 patients with DLBCL who experienced relapse or whose condition was refractory to prior therapies. About one-third of patients (30%) had received prior CAR T-cell therapy. Additionally, for 83% of patients, the condition was refractory to their most recent therapy.

Glofitamab was given to all patients as a fixed course for 8.5 months.

More than half (56%) achieved an overall response, and 43% achieved a complete response. Over two-thirds (68.5%) of those who responded continued to respond for at least 9 months The median duration of response was 1.5 years.

The most common adverse events were cytokine release syndrome (CRS; 70%), which may be serious or life-threatening; musculoskeletal pain (21%); fatigue (20%); and rash (20%). CRS was generally of low grade (52% of patients experienced grade 1 CRS, and 14% experienced grade 2).

Results from the NP30179 trial were published in December 2022.

The complete response rates seen with glofitamab rivals the durable complete response that has been observed with CAR T-cell therapy, Dr. Bartlett noted in the accompanying editorial. “Although these results are promising, it is still too early to estimate the curative potential of glofitamab.”

The Food and Drug Administration has granted accelerated approval for glofitamab (Columvi) for use in certain types of lymphoma.

The indication is for use in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified or with LBCL arising from follicular lymphoma who have received two or more lines of systemic therapy.

The product is a T cell–engaging bispecific antibody developed by Genentech, which has a similar product, mosunetuzumab-axgb (Lunsumio), for the treatment of follicular lymphoma. Lunsumio was approved in December 2022.

These drugs could be considered a first choice in the setting of third-line therapy, suggests an expert writing recently in The New England Journal of Medicine.

Nancy Bartlett, MD, from the Siteman Cancer Center, Washington University in St. Louis, is the author of an editorial that accompanied the publication of results with glofitamab in the pivotal trial that led to its approval.

“Bispecific agents will be an excellent option for the 60% of patients in whom second-line CAR [chimeric antigen receptor] T-cell therapy fails,” she wrote in her editorial.

Dr. Bartlett suggests that these agents may be preferred over CAR T cells. “If longer follow-up confirms that the majority of complete remissions with bispecific agents are durable, on the basis of the advantages of availability (including in the community setting) and more favorable immediate and late toxic-effect profiles, bispecific agents could be considered as the initial choice. ... CAR T-cell therapy could be held in reserve for patients who do not have a complete response or who have a relapse after a complete response.”
 

Most common form of non-Hodgkin’s lymphoma

DLBCL is the most common form of non-Hodgkin’s lymphoma in the United States, the company noted in a press release. While many people with DLBCL are responsive to treatment, the majority of those who experience relapse or whose condition is refractory to subsequent treatments have poor outcomes.

“Patients with relapsed or refractory diffuse large B-cell lymphoma may experience rapid progression of their cancer and often urgently need an effective treatment option that can be administered without delay,” commented Krish Patel, MD, director of the lymphoma program at the Swedish Cancer Institute in Seattle, who is an investigator on the clinical trial that led to the product’s approval. He said that the results from trials suggest that glofitamab gives patients “a chance for complete remission with a fixed-duration immunotherapy and that such remissions can potentially be sustained after the end of their treatment.”

The accelerated approval is based on response rate and durability of response results from the phase 1/2 NP30179 study.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

This trial involved 132 patients with DLBCL who experienced relapse or whose condition was refractory to prior therapies. About one-third of patients (30%) had received prior CAR T-cell therapy. Additionally, for 83% of patients, the condition was refractory to their most recent therapy.

Glofitamab was given to all patients as a fixed course for 8.5 months.

More than half (56%) achieved an overall response, and 43% achieved a complete response. Over two-thirds (68.5%) of those who responded continued to respond for at least 9 months The median duration of response was 1.5 years.

The most common adverse events were cytokine release syndrome (CRS; 70%), which may be serious or life-threatening; musculoskeletal pain (21%); fatigue (20%); and rash (20%). CRS was generally of low grade (52% of patients experienced grade 1 CRS, and 14% experienced grade 2).

Results from the NP30179 trial were published in December 2022.

The complete response rates seen with glofitamab rivals the durable complete response that has been observed with CAR T-cell therapy, Dr. Bartlett noted in the accompanying editorial. “Although these results are promising, it is still too early to estimate the curative potential of glofitamab.”

The Food and Drug Administration has granted accelerated approval for glofitamab (Columvi) for use in certain types of lymphoma.

The indication is for use in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified or with LBCL arising from follicular lymphoma who have received two or more lines of systemic therapy.

The product is a T cell–engaging bispecific antibody developed by Genentech, which has a similar product, mosunetuzumab-axgb (Lunsumio), for the treatment of follicular lymphoma. Lunsumio was approved in December 2022.

These drugs could be considered a first choice in the setting of third-line therapy, suggests an expert writing recently in The New England Journal of Medicine.

Nancy Bartlett, MD, from the Siteman Cancer Center, Washington University in St. Louis, is the author of an editorial that accompanied the publication of results with glofitamab in the pivotal trial that led to its approval.

“Bispecific agents will be an excellent option for the 60% of patients in whom second-line CAR [chimeric antigen receptor] T-cell therapy fails,” she wrote in her editorial.

Dr. Bartlett suggests that these agents may be preferred over CAR T cells. “If longer follow-up confirms that the majority of complete remissions with bispecific agents are durable, on the basis of the advantages of availability (including in the community setting) and more favorable immediate and late toxic-effect profiles, bispecific agents could be considered as the initial choice. ... CAR T-cell therapy could be held in reserve for patients who do not have a complete response or who have a relapse after a complete response.”
 

Most common form of non-Hodgkin’s lymphoma

DLBCL is the most common form of non-Hodgkin’s lymphoma in the United States, the company noted in a press release. While many people with DLBCL are responsive to treatment, the majority of those who experience relapse or whose condition is refractory to subsequent treatments have poor outcomes.

“Patients with relapsed or refractory diffuse large B-cell lymphoma may experience rapid progression of their cancer and often urgently need an effective treatment option that can be administered without delay,” commented Krish Patel, MD, director of the lymphoma program at the Swedish Cancer Institute in Seattle, who is an investigator on the clinical trial that led to the product’s approval. He said that the results from trials suggest that glofitamab gives patients “a chance for complete remission with a fixed-duration immunotherapy and that such remissions can potentially be sustained after the end of their treatment.”

The accelerated approval is based on response rate and durability of response results from the phase 1/2 NP30179 study.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

This trial involved 132 patients with DLBCL who experienced relapse or whose condition was refractory to prior therapies. About one-third of patients (30%) had received prior CAR T-cell therapy. Additionally, for 83% of patients, the condition was refractory to their most recent therapy.

Glofitamab was given to all patients as a fixed course for 8.5 months.

More than half (56%) achieved an overall response, and 43% achieved a complete response. Over two-thirds (68.5%) of those who responded continued to respond for at least 9 months The median duration of response was 1.5 years.

The most common adverse events were cytokine release syndrome (CRS; 70%), which may be serious or life-threatening; musculoskeletal pain (21%); fatigue (20%); and rash (20%). CRS was generally of low grade (52% of patients experienced grade 1 CRS, and 14% experienced grade 2).

Results from the NP30179 trial were published in December 2022.

The complete response rates seen with glofitamab rivals the durable complete response that has been observed with CAR T-cell therapy, Dr. Bartlett noted in the accompanying editorial. “Although these results are promising, it is still too early to estimate the curative potential of glofitamab.”

Members of a Food and Drug Administration advisory committee have unanimously concluded that a postmarketing study confirms the benefit of the Alzheimer’s drug lecanemab (Leqembi, Eisai), paving the way for traditional approval.

“Overall, the study demonstrated clearly that this is an effective treatment,” said acting chair Robert C. Alexander, MD, chief scientific officer, Alzheimer’s Prevention Initiative, Banner Alzheimer’s Institute, and research professor, department of psychiatry, University of Arizona, Phoenix, during the meeting.

An intravenous infusion targeting amyloid-beta, lecanemab received accelerated FDA approved earlier in 2023 for the treatment of early Alzheimer’s disease (AD). The company was required to complete a confirmatory study to verify and describe the product’s clinical benefit.

The Peripheral and Central Nervous System Drugs Advisory Committee met to discuss this phase 3 study (CLARITY-AD). The multicenter, double-blind study included 1,795 patients (mean age, 71 years) who had mild cognitive impairment caused by AD or mild AD dementia.
 

Delayed progression

Study participants had a broad range of comorbidities, and many were concomitantly receiving other medications. Black people were underrepresented in the study at just 3% of the total cohort.

Patients were randomly assigned to receive placebo or lecanemab 10 mg/kg biweekly. In addition to a placebo-controlled period and safety follow-up, the study has an ongoing extension phase of up to 4 years.

The study met its primary endpoint, showing a highly statistically significant 27% less decline on the Clinical Dementia Rating-Sum of Boxes at 18 months (difference in adjusted mean, –0.45; 95% CI, –0.67 to –0.23; P = .00005).

This was supported by a significant 26% difference on the AD Assessment Scale–Cognitive Subscale with 14 tasks (ADAS-Cog 14).

The drug also affected function, with a 37% decrease, compared with placebo, on the AD Cooperative Study–Activities of Daily Living Scale for Mild Cognitive Impairment.

Committee members heard that the results signal delays in disease progression by about 5 months, giving patients more time to live independently and participate in hobbies and interests.

Patients who received the active drug also experienced quality of life benefits. Compared with patients who received placebo, those who took lecanemab had 49% less decline as measured with the European Quality of Life–5 Dimensions scale and 56% less decline as measured by the Quality of Life in AD scale, and caregivers reported less burden.

Lecanemab also affected biomarkers of amyloid, tau, and neurodegeneration, providing a biological basis for the treatment effects consistent with slowing of disease progression.
 

Unanimous support

All six committee members agreed by vote that the study provides evidence of clinical benefit. They variously descried the study and results as “robust,” “compelling,” “well conducted,” “clear and consistent,” and “clinically meaningful.”

In the active treatment group, there was a higher incidence of amyloid-related imaging abnormalities (ARIAs), which can be serious and life-threatening but are usually asymptomatic. In this study, most ARIAs had resolved by 3 months.

Deaths occurred in 0.8% of the placebo and 0.7% of the treatment group. Dean Follmann, PhD, assistant director for biostatistics, National Institute of Allergy and Infectious Diseases, Bethesda, Md., noted that the numbers of deaths and serious adverse events were “quite similar” in the two groups.

“And for serious ARIA, there was an imbalance favoring placebo, but overall, these were pretty rare,” he said.
 

Subgroup concerns

Committee members discussed the risk/benefit profile for three subgroups of patients – those with apolipoprotein E4 (apo E4) allele, patients taking an anticoagulant, and those with cerebral amyloid angiopathy (CAA).

In the apo E4 group, the study’s primary endpoint did not favor the drug, but secondary endpoints did.

“I think the general feeling [for apo E4 status] is that the risk/benefit still remains favorable, especially when looking across multiple endpoints,” said Dr. Alexander.

However, some members supported recommending genetic testing before initiating the drug.

The views were more diverse for the use of lecanemab in the presence of an anticoagulant, which may increase the risk for cerebral hemorrhage. Some committee members strongly recommended that these patients not receive lecanemab, while others highlighted the need for more information, owing to uncertainties about the risks.

With respect to CAA, most members supported the idea of considering use of the drug in the presence of this condition, but only after discussing the risks with patients and their families and in the presence of a robust reporting system.

An Alzheimer’s Association representative was in attendance during the public hearing portion of the meeting to express support for traditional approval of lecanemab for people with early AD.

The association strongly favors full Medicare coverage for FDA-approved AD treatments. The Centers for Medicare & Medicaid Services has determined that AD treatments receiving traditional FDA approval will be covered if clinicians register and enter data in a registry.

“While this is an important signal that CMS wants to improve access to FDA-approved treatments, registry as a condition of coverage is an unnecessary and potentially harmful barrier,” said the Alzheimer’s Association in a press release following the meeting.

A version of this article first appeared on Medscape.com.

Members of a Food and Drug Administration advisory committee have unanimously concluded that a postmarketing study confirms the benefit of the Alzheimer’s drug lecanemab (Leqembi, Eisai), paving the way for traditional approval.

“Overall, the study demonstrated clearly that this is an effective treatment,” said acting chair Robert C. Alexander, MD, chief scientific officer, Alzheimer’s Prevention Initiative, Banner Alzheimer’s Institute, and research professor, department of psychiatry, University of Arizona, Phoenix, during the meeting.

An intravenous infusion targeting amyloid-beta, lecanemab received accelerated FDA approved earlier in 2023 for the treatment of early Alzheimer’s disease (AD). The company was required to complete a confirmatory study to verify and describe the product’s clinical benefit.

The Peripheral and Central Nervous System Drugs Advisory Committee met to discuss this phase 3 study (CLARITY-AD). The multicenter, double-blind study included 1,795 patients (mean age, 71 years) who had mild cognitive impairment caused by AD or mild AD dementia.
 

Delayed progression

Study participants had a broad range of comorbidities, and many were concomitantly receiving other medications. Black people were underrepresented in the study at just 3% of the total cohort.

Patients were randomly assigned to receive placebo or lecanemab 10 mg/kg biweekly. In addition to a placebo-controlled period and safety follow-up, the study has an ongoing extension phase of up to 4 years.

The study met its primary endpoint, showing a highly statistically significant 27% less decline on the Clinical Dementia Rating-Sum of Boxes at 18 months (difference in adjusted mean, –0.45; 95% CI, –0.67 to –0.23; P = .00005).

This was supported by a significant 26% difference on the AD Assessment Scale–Cognitive Subscale with 14 tasks (ADAS-Cog 14).

The drug also affected function, with a 37% decrease, compared with placebo, on the AD Cooperative Study–Activities of Daily Living Scale for Mild Cognitive Impairment.

Committee members heard that the results signal delays in disease progression by about 5 months, giving patients more time to live independently and participate in hobbies and interests.

Patients who received the active drug also experienced quality of life benefits. Compared with patients who received placebo, those who took lecanemab had 49% less decline as measured with the European Quality of Life–5 Dimensions scale and 56% less decline as measured by the Quality of Life in AD scale, and caregivers reported less burden.

Lecanemab also affected biomarkers of amyloid, tau, and neurodegeneration, providing a biological basis for the treatment effects consistent with slowing of disease progression.
 

Unanimous support

All six committee members agreed by vote that the study provides evidence of clinical benefit. They variously descried the study and results as “robust,” “compelling,” “well conducted,” “clear and consistent,” and “clinically meaningful.”

In the active treatment group, there was a higher incidence of amyloid-related imaging abnormalities (ARIAs), which can be serious and life-threatening but are usually asymptomatic. In this study, most ARIAs had resolved by 3 months.

Deaths occurred in 0.8% of the placebo and 0.7% of the treatment group. Dean Follmann, PhD, assistant director for biostatistics, National Institute of Allergy and Infectious Diseases, Bethesda, Md., noted that the numbers of deaths and serious adverse events were “quite similar” in the two groups.

“And for serious ARIA, there was an imbalance favoring placebo, but overall, these were pretty rare,” he said.
 

Subgroup concerns

Committee members discussed the risk/benefit profile for three subgroups of patients – those with apolipoprotein E4 (apo E4) allele, patients taking an anticoagulant, and those with cerebral amyloid angiopathy (CAA).

In the apo E4 group, the study’s primary endpoint did not favor the drug, but secondary endpoints did.

“I think the general feeling [for apo E4 status] is that the risk/benefit still remains favorable, especially when looking across multiple endpoints,” said Dr. Alexander.

However, some members supported recommending genetic testing before initiating the drug.

The views were more diverse for the use of lecanemab in the presence of an anticoagulant, which may increase the risk for cerebral hemorrhage. Some committee members strongly recommended that these patients not receive lecanemab, while others highlighted the need for more information, owing to uncertainties about the risks.

With respect to CAA, most members supported the idea of considering use of the drug in the presence of this condition, but only after discussing the risks with patients and their families and in the presence of a robust reporting system.

An Alzheimer’s Association representative was in attendance during the public hearing portion of the meeting to express support for traditional approval of lecanemab for people with early AD.

The association strongly favors full Medicare coverage for FDA-approved AD treatments. The Centers for Medicare & Medicaid Services has determined that AD treatments receiving traditional FDA approval will be covered if clinicians register and enter data in a registry.

“While this is an important signal that CMS wants to improve access to FDA-approved treatments, registry as a condition of coverage is an unnecessary and potentially harmful barrier,” said the Alzheimer’s Association in a press release following the meeting.

A version of this article first appeared on Medscape.com.

Members of a Food and Drug Administration advisory committee have unanimously concluded that a postmarketing study confirms the benefit of the Alzheimer’s drug lecanemab (Leqembi, Eisai), paving the way for traditional approval.

“Overall, the study demonstrated clearly that this is an effective treatment,” said acting chair Robert C. Alexander, MD, chief scientific officer, Alzheimer’s Prevention Initiative, Banner Alzheimer’s Institute, and research professor, department of psychiatry, University of Arizona, Phoenix, during the meeting.

An intravenous infusion targeting amyloid-beta, lecanemab received accelerated FDA approved earlier in 2023 for the treatment of early Alzheimer’s disease (AD). The company was required to complete a confirmatory study to verify and describe the product’s clinical benefit.

The Peripheral and Central Nervous System Drugs Advisory Committee met to discuss this phase 3 study (CLARITY-AD). The multicenter, double-blind study included 1,795 patients (mean age, 71 years) who had mild cognitive impairment caused by AD or mild AD dementia.
 

Delayed progression

Study participants had a broad range of comorbidities, and many were concomitantly receiving other medications. Black people were underrepresented in the study at just 3% of the total cohort.

Patients were randomly assigned to receive placebo or lecanemab 10 mg/kg biweekly. In addition to a placebo-controlled period and safety follow-up, the study has an ongoing extension phase of up to 4 years.

The study met its primary endpoint, showing a highly statistically significant 27% less decline on the Clinical Dementia Rating-Sum of Boxes at 18 months (difference in adjusted mean, –0.45; 95% CI, –0.67 to –0.23; P = .00005).

This was supported by a significant 26% difference on the AD Assessment Scale–Cognitive Subscale with 14 tasks (ADAS-Cog 14).

The drug also affected function, with a 37% decrease, compared with placebo, on the AD Cooperative Study–Activities of Daily Living Scale for Mild Cognitive Impairment.

Committee members heard that the results signal delays in disease progression by about 5 months, giving patients more time to live independently and participate in hobbies and interests.

Patients who received the active drug also experienced quality of life benefits. Compared with patients who received placebo, those who took lecanemab had 49% less decline as measured with the European Quality of Life–5 Dimensions scale and 56% less decline as measured by the Quality of Life in AD scale, and caregivers reported less burden.

Lecanemab also affected biomarkers of amyloid, tau, and neurodegeneration, providing a biological basis for the treatment effects consistent with slowing of disease progression.
 

Unanimous support

All six committee members agreed by vote that the study provides evidence of clinical benefit. They variously descried the study and results as “robust,” “compelling,” “well conducted,” “clear and consistent,” and “clinically meaningful.”

In the active treatment group, there was a higher incidence of amyloid-related imaging abnormalities (ARIAs), which can be serious and life-threatening but are usually asymptomatic. In this study, most ARIAs had resolved by 3 months.

Deaths occurred in 0.8% of the placebo and 0.7% of the treatment group. Dean Follmann, PhD, assistant director for biostatistics, National Institute of Allergy and Infectious Diseases, Bethesda, Md., noted that the numbers of deaths and serious adverse events were “quite similar” in the two groups.

“And for serious ARIA, there was an imbalance favoring placebo, but overall, these were pretty rare,” he said.
 

Subgroup concerns

Committee members discussed the risk/benefit profile for three subgroups of patients – those with apolipoprotein E4 (apo E4) allele, patients taking an anticoagulant, and those with cerebral amyloid angiopathy (CAA).

In the apo E4 group, the study’s primary endpoint did not favor the drug, but secondary endpoints did.

“I think the general feeling [for apo E4 status] is that the risk/benefit still remains favorable, especially when looking across multiple endpoints,” said Dr. Alexander.

However, some members supported recommending genetic testing before initiating the drug.

The views were more diverse for the use of lecanemab in the presence of an anticoagulant, which may increase the risk for cerebral hemorrhage. Some committee members strongly recommended that these patients not receive lecanemab, while others highlighted the need for more information, owing to uncertainties about the risks.

With respect to CAA, most members supported the idea of considering use of the drug in the presence of this condition, but only after discussing the risks with patients and their families and in the presence of a robust reporting system.

An Alzheimer’s Association representative was in attendance during the public hearing portion of the meeting to express support for traditional approval of lecanemab for people with early AD.

The association strongly favors full Medicare coverage for FDA-approved AD treatments. The Centers for Medicare & Medicaid Services has determined that AD treatments receiving traditional FDA approval will be covered if clinicians register and enter data in a registry.

“While this is an important signal that CMS wants to improve access to FDA-approved treatments, registry as a condition of coverage is an unnecessary and potentially harmful barrier,” said the Alzheimer’s Association in a press release following the meeting.

A version of this article first appeared on Medscape.com.

The Centers for Medicare & Medicaid Services has announced that Medicare will cover drugs designed to slow Alzheimer’s disease once they receive traditional approval by the Food and Drug Administration.

The one caveat is that CMS will require physicians to participate in registries that collect evidence about how these drugs work in the real world.

Physicians will be able to submit this evidence through a nationwide, CMS-facilitated portal that will be available when any product gains traditional approval and will collect information via an easy-to-use format.

“If the FDA grants traditional approval, then Medicare will cover it in appropriate settings that also support the collection of real-world information to study the usefulness of these drugs for people with Medicare,” the CMS says in a news release.

“CMS has always been committed to helping people obtain timely access to innovative treatments that meaningfully improve care and outcomes for this disease,” added CMS Administrator Chiquita Brooks-LaSure.

“If the FDA grants traditional approval, CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered,” Ms. Brooks-LaSure explained.

The CMS says broader Medicare coverage for an Alzheimer’s drug would begin on the same day the FDA grants traditional approval. Under CMS’ current coverage policy, if the FDA grants traditional approval to other drugs in this class, they would also be eligible for broader coverage.

Currently two drugs in this class – aducanumab (Aduhelm) and lecanemab (Leqembi) – have received accelerated approval from the FDA, but no product has received traditional approval.

Lecanemab might be the first to cross the line.

On June 9, the FDA Peripheral and Central Nervous System Drugs Advisory Committee will discuss results of a confirmatory trial of lecanemab, with a potential decision on traditional approval expected shortly thereafter.

A version of this article first appeared on Medscape.com.

The Centers for Medicare & Medicaid Services has announced that Medicare will cover drugs designed to slow Alzheimer’s disease once they receive traditional approval by the Food and Drug Administration.

The one caveat is that CMS will require physicians to participate in registries that collect evidence about how these drugs work in the real world.

Physicians will be able to submit this evidence through a nationwide, CMS-facilitated portal that will be available when any product gains traditional approval and will collect information via an easy-to-use format.

“If the FDA grants traditional approval, then Medicare will cover it in appropriate settings that also support the collection of real-world information to study the usefulness of these drugs for people with Medicare,” the CMS says in a news release.

“CMS has always been committed to helping people obtain timely access to innovative treatments that meaningfully improve care and outcomes for this disease,” added CMS Administrator Chiquita Brooks-LaSure.

“If the FDA grants traditional approval, CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered,” Ms. Brooks-LaSure explained.

The CMS says broader Medicare coverage for an Alzheimer’s drug would begin on the same day the FDA grants traditional approval. Under CMS’ current coverage policy, if the FDA grants traditional approval to other drugs in this class, they would also be eligible for broader coverage.

Currently two drugs in this class – aducanumab (Aduhelm) and lecanemab (Leqembi) – have received accelerated approval from the FDA, but no product has received traditional approval.

Lecanemab might be the first to cross the line.

On June 9, the FDA Peripheral and Central Nervous System Drugs Advisory Committee will discuss results of a confirmatory trial of lecanemab, with a potential decision on traditional approval expected shortly thereafter.

A version of this article first appeared on Medscape.com.

The Centers for Medicare & Medicaid Services has announced that Medicare will cover drugs designed to slow Alzheimer’s disease once they receive traditional approval by the Food and Drug Administration.

The one caveat is that CMS will require physicians to participate in registries that collect evidence about how these drugs work in the real world.

Physicians will be able to submit this evidence through a nationwide, CMS-facilitated portal that will be available when any product gains traditional approval and will collect information via an easy-to-use format.

“If the FDA grants traditional approval, then Medicare will cover it in appropriate settings that also support the collection of real-world information to study the usefulness of these drugs for people with Medicare,” the CMS says in a news release.

“CMS has always been committed to helping people obtain timely access to innovative treatments that meaningfully improve care and outcomes for this disease,” added CMS Administrator Chiquita Brooks-LaSure.

“If the FDA grants traditional approval, CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered,” Ms. Brooks-LaSure explained.

The CMS says broader Medicare coverage for an Alzheimer’s drug would begin on the same day the FDA grants traditional approval. Under CMS’ current coverage policy, if the FDA grants traditional approval to other drugs in this class, they would also be eligible for broader coverage.

Currently two drugs in this class – aducanumab (Aduhelm) and lecanemab (Leqembi) – have received accelerated approval from the FDA, but no product has received traditional approval.

Lecanemab might be the first to cross the line.

On June 9, the FDA Peripheral and Central Nervous System Drugs Advisory Committee will discuss results of a confirmatory trial of lecanemab, with a potential decision on traditional approval expected shortly thereafter.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved extended-release injection buprenorphine (Brixadi, Braeburn) for the treatment of moderate to severe opioid use disorder (OUD).

Olivier Le Moal/Getty Images

The medication comes in two formulations: a weekly and a monthly version. The weekly treatment is indicated in patients who have initiated treatment with a single dose of transmucosal buprenorphine or who are already being treated with the drug. The monthly version is for patients already receiving buprenorphine.

“Buprenorphine is an important treatment option for opioid use disorder. Today’s approval expands dosing options and provides people with opioid use disorder a greater opportunity to sustain long-term recovery,” said FDA Commissioner Robert M. Califf, MD, in a release. “The FDA will continue to take the critical steps necessary to pursue efforts that advance evidence-based treatments for substance use disorders, which is a strategic priority under the FDA’s Overdose Prevention Framework,” Dr. Califf added.

The Substance Abuse and Mental Health Services Administration reports that patients receiving medication for OUD have their risk for all-cause mortality cut by 50%.

In its release, the FDA said that it remains committed to increasing treatment options for OUD. Earlier this month, the agency issued a joint letter with SAMHSA to underscore the importance of counseling and other services as part of a comprehensive treatment plan the disorder. It also emphasized that receiving buprenorphine should not be contingent on participating in such services.

Brixadi is approved in both weekly and monthly subcutaneous injectable formulations at varying doses, including lower doses that may be appropriate for patients who do not tolerate higher doses of extended-release buprenorphine that are currently available.

The drug will be available through a Risk Evaluation and Mitigation Strategy program and administered only by health care providers in a health care setting.

The most common adverse reactions associated with the drug include injection-site pain, headache, constipation, nausea, injection-site erythema, injection-site pruritus, insomnia, and urinary tract infections. The FDA reports that such side effects occur in at least 5% of patients.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved extended-release injection buprenorphine (Brixadi, Braeburn) for the treatment of moderate to severe opioid use disorder (OUD).

Olivier Le Moal/Getty Images

The medication comes in two formulations: a weekly and a monthly version. The weekly treatment is indicated in patients who have initiated treatment with a single dose of transmucosal buprenorphine or who are already being treated with the drug. The monthly version is for patients already receiving buprenorphine.

“Buprenorphine is an important treatment option for opioid use disorder. Today’s approval expands dosing options and provides people with opioid use disorder a greater opportunity to sustain long-term recovery,” said FDA Commissioner Robert M. Califf, MD, in a release. “The FDA will continue to take the critical steps necessary to pursue efforts that advance evidence-based treatments for substance use disorders, which is a strategic priority under the FDA’s Overdose Prevention Framework,” Dr. Califf added.

The Substance Abuse and Mental Health Services Administration reports that patients receiving medication for OUD have their risk for all-cause mortality cut by 50%.

In its release, the FDA said that it remains committed to increasing treatment options for OUD. Earlier this month, the agency issued a joint letter with SAMHSA to underscore the importance of counseling and other services as part of a comprehensive treatment plan the disorder. It also emphasized that receiving buprenorphine should not be contingent on participating in such services.

Brixadi is approved in both weekly and monthly subcutaneous injectable formulations at varying doses, including lower doses that may be appropriate for patients who do not tolerate higher doses of extended-release buprenorphine that are currently available.

The drug will be available through a Risk Evaluation and Mitigation Strategy program and administered only by health care providers in a health care setting.

The most common adverse reactions associated with the drug include injection-site pain, headache, constipation, nausea, injection-site erythema, injection-site pruritus, insomnia, and urinary tract infections. The FDA reports that such side effects occur in at least 5% of patients.

A version of this article originally appeared on Medscape.com.

The Food and Drug Administration has approved extended-release injection buprenorphine (Brixadi, Braeburn) for the treatment of moderate to severe opioid use disorder (OUD).

Olivier Le Moal/Getty Images

The medication comes in two formulations: a weekly and a monthly version. The weekly treatment is indicated in patients who have initiated treatment with a single dose of transmucosal buprenorphine or who are already being treated with the drug. The monthly version is for patients already receiving buprenorphine.

“Buprenorphine is an important treatment option for opioid use disorder. Today’s approval expands dosing options and provides people with opioid use disorder a greater opportunity to sustain long-term recovery,” said FDA Commissioner Robert M. Califf, MD, in a release. “The FDA will continue to take the critical steps necessary to pursue efforts that advance evidence-based treatments for substance use disorders, which is a strategic priority under the FDA’s Overdose Prevention Framework,” Dr. Califf added.

The Substance Abuse and Mental Health Services Administration reports that patients receiving medication for OUD have their risk for all-cause mortality cut by 50%.

In its release, the FDA said that it remains committed to increasing treatment options for OUD. Earlier this month, the agency issued a joint letter with SAMHSA to underscore the importance of counseling and other services as part of a comprehensive treatment plan the disorder. It also emphasized that receiving buprenorphine should not be contingent on participating in such services.

Brixadi is approved in both weekly and monthly subcutaneous injectable formulations at varying doses, including lower doses that may be appropriate for patients who do not tolerate higher doses of extended-release buprenorphine that are currently available.

The drug will be available through a Risk Evaluation and Mitigation Strategy program and administered only by health care providers in a health care setting.

The most common adverse reactions associated with the drug include injection-site pain, headache, constipation, nausea, injection-site erythema, injection-site pruritus, insomnia, and urinary tract infections. The FDA reports that such side effects occur in at least 5% of patients.

A version of this article originally appeared on Medscape.com.