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Belimumab Autoinjector Approved for Pediatric Lupus
The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.
Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.
“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement.
An estimated 5000-10,000 children in the United States are living with SLE.
Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.
The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg.
The autoinjector “will be available immediately” for caregivers, the company announcement said.
“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.
Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.
“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement.
An estimated 5000-10,000 children in the United States are living with SLE.
Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.
The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg.
The autoinjector “will be available immediately” for caregivers, the company announcement said.
“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.
Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.
“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement.
An estimated 5000-10,000 children in the United States are living with SLE.
Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.
The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg.
The autoinjector “will be available immediately” for caregivers, the company announcement said.
“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”
A version of this article appeared on Medscape.com.
FDA Approves Tarlatamab for Extensive-Stage Small Cell Lung Cancer
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
FDA OKs First Multitarget Stool RNA Test for CRC Screening
ColoSense, which had breakthrough device designation by the FDA, detects colorectal neoplasia–associated RNA markers and the presence of occult hemoglobin in human stool.
A positive ColoSense test result may indicate the presence of CRC, advanced adenomas, or serrated precancerous lesions and should be followed by a colonoscopy, the company said in a news release.
The FDA approval was based on results of the CRC-PREVENT trial, which evaluated the ColoSense mt-sRNA test in a diverse group of adults undergoing colonoscopy. 
The mt-sRNA test results were compared with the colonoscopy results.
Among all average-risk individuals, the sensitivity of the mt-sRNA test was 93% for CRC, 100% for early (stage I) CRC, and 45% for advanced adenomas. In a subgroup of those aged 45-49 years, the sensitivity was 100% for CRC and 44% for advanced adenomas.
The trial results were presented last year at the American College of Gastroenterology annual meeting and simultaneously published in JAMA .
CRC is the second deadliest cancer in the United States, and adherence rates to recommended colonoscopies as a screening modality have remained consistently low at roughly 60%.
Cases of CRC are also rising among people younger than age 50 years, leading the United States Preventive Services Task Force to recommend initiation of CRC screening at age 45 years.
“The growing number of adults diagnosed with colorectal cancer underscores the urgent need for innovative approaches in screening. It’s essential to eliminate obstacles and broaden the availability of screening methods for healthcare providers and patients,” Anjee Davis, president of Fight CRC, said in the news release.
“We hope that introducing new FDA-approved diagnostic tools, including stool-based tests like ColoSense, will help to advance access and increase screening rates, ultimately reducing the impact of late-stage colorectal cancer diagnoses,” Ms. Davis said.
The company plans to make ColoSense available in the United States later this year or early in 2025.
A version of this article appeared on Medscape.com.
ColoSense, which had breakthrough device designation by the FDA, detects colorectal neoplasia–associated RNA markers and the presence of occult hemoglobin in human stool.
A positive ColoSense test result may indicate the presence of CRC, advanced adenomas, or serrated precancerous lesions and should be followed by a colonoscopy, the company said in a news release.
The FDA approval was based on results of the CRC-PREVENT trial, which evaluated the ColoSense mt-sRNA test in a diverse group of adults undergoing colonoscopy.
The mt-sRNA test results were compared with the colonoscopy results.
Among all average-risk individuals, the sensitivity of the mt-sRNA test was 93% for CRC, 100% for early (stage I) CRC, and 45% for advanced adenomas. In a subgroup of those aged 45-49 years, the sensitivity was 100% for CRC and 44% for advanced adenomas.
The trial results were presented last year at the American College of Gastroenterology annual meeting and simultaneously published in JAMA .
CRC is the second deadliest cancer in the United States, and adherence rates to recommended colonoscopies as a screening modality have remained consistently low at roughly 60%.
Cases of CRC are also rising among people younger than age 50 years, leading the United States Preventive Services Task Force to recommend initiation of CRC screening at age 45 years.
“The growing number of adults diagnosed with colorectal cancer underscores the urgent need for innovative approaches in screening. It’s essential to eliminate obstacles and broaden the availability of screening methods for healthcare providers and patients,” Anjee Davis, president of Fight CRC, said in the news release.
“We hope that introducing new FDA-approved diagnostic tools, including stool-based tests like ColoSense, will help to advance access and increase screening rates, ultimately reducing the impact of late-stage colorectal cancer diagnoses,” Ms. Davis said.
The company plans to make ColoSense available in the United States later this year or early in 2025.
A version of this article appeared on Medscape.com.
ColoSense, which had breakthrough device designation by the FDA, detects colorectal neoplasia–associated RNA markers and the presence of occult hemoglobin in human stool.
A positive ColoSense test result may indicate the presence of CRC, advanced adenomas, or serrated precancerous lesions and should be followed by a colonoscopy, the company said in a news release.
The FDA approval was based on results of the CRC-PREVENT trial, which evaluated the ColoSense mt-sRNA test in a diverse group of adults undergoing colonoscopy.
The mt-sRNA test results were compared with the colonoscopy results.
Among all average-risk individuals, the sensitivity of the mt-sRNA test was 93% for CRC, 100% for early (stage I) CRC, and 45% for advanced adenomas. In a subgroup of those aged 45-49 years, the sensitivity was 100% for CRC and 44% for advanced adenomas.
The trial results were presented last year at the American College of Gastroenterology annual meeting and simultaneously published in JAMA .
CRC is the second deadliest cancer in the United States, and adherence rates to recommended colonoscopies as a screening modality have remained consistently low at roughly 60%.
Cases of CRC are also rising among people younger than age 50 years, leading the United States Preventive Services Task Force to recommend initiation of CRC screening at age 45 years.
“The growing number of adults diagnosed with colorectal cancer underscores the urgent need for innovative approaches in screening. It’s essential to eliminate obstacles and broaden the availability of screening methods for healthcare providers and patients,” Anjee Davis, president of Fight CRC, said in the news release.
“We hope that introducing new FDA-approved diagnostic tools, including stool-based tests like ColoSense, will help to advance access and increase screening rates, ultimately reducing the impact of late-stage colorectal cancer diagnoses,” Ms. Davis said.
The company plans to make ColoSense available in the United States later this year or early in 2025.
A version of this article appeared on Medscape.com.
FDA Approves New Bladder Cancer Drug
Specifically, the agent is approved to treat patients with BCG-unresponsive non–muscle-invasive bladder cancer carcinoma in situ with or without Ta or T1 papillary disease.
The FDA declined an initial approval for the combination in May 2023 because of deficiencies the agency observed during its prelicense inspection of third-party manufacturing organizations. In October 2023, ImmunityBio resubmitted the Biologics License Application, which was accepted.
The new therapy represents addresses “an unmet need” in this high-risk bladder cancer population, the company stated in a press release announcing the initial study findings. Typically, patients with intermediate or high-risk disease undergo bladder tumor resection followed by treatment with BCG, but the cancer recurs in up to 50% of patients, including those who experience a complete response, explained ImmunityBio, which acquired Altor BioScience.
Approval was based on findings from the single arm, phase 2/3 open-label QUILT-3.032 study, which included 77 patients with BCG-unresponsive, high-risk disease following transurethral resection. All had Eastern Cooperative Oncology Group performance status of 0-2.
Patients received nogapendekin alfa inbakicept-pmln induction via intravesical instillation with BCG followed by maintenance therapy for up to 37 months.
According to the FDA’s press release, 62% of patients had a complete response, defined as a negative cystoscopy and urine cytology; 58% of those with a complete response had a duration of response lasting at least 12 months and 40% had a duration of response lasting 24 months or longer.
The safety of the combination was evaluated in a cohort of 88 patients. Serious adverse reactions occurred in 16% of patients. The most common treatment-emergent adverse effects included dysuria, pollakiuria, and hematuria, which are associated with intravesical BCG; 86% of these events were grade 1 or 2. Overall, 7% of patients discontinued the combination owing to adverse reactions.
The recommended dose is 400 mcg administered intravesically with BCG once a week for 6 weeks as induction therapy, with an option for a second induction course if patients don’t achieve a complete response at 3 months. The recommended maintenance therapy dose is 400 mcg with BCG once a week for 3 weeks at months 4, 7, 10, 13, and 19. Patients who achieve a complete response at 25 months and beyond may receive maintenance instillations with BCG once a week for 3 weeks at months 25, 31, and 37. The maximum treatment duration is 37 months.
The FDA recommends discontinuing treatment if disease persists after second induction or owing to disease recurrence, progression, or unacceptable toxicity.
A version of this article appeared on Medscape.com.
Specifically, the agent is approved to treat patients with BCG-unresponsive non–muscle-invasive bladder cancer carcinoma in situ with or without Ta or T1 papillary disease.
The FDA declined an initial approval for the combination in May 2023 because of deficiencies the agency observed during its prelicense inspection of third-party manufacturing organizations. In October 2023, ImmunityBio resubmitted the Biologics License Application, which was accepted.
The new therapy represents addresses “an unmet need” in this high-risk bladder cancer population, the company stated in a press release announcing the initial study findings. Typically, patients with intermediate or high-risk disease undergo bladder tumor resection followed by treatment with BCG, but the cancer recurs in up to 50% of patients, including those who experience a complete response, explained ImmunityBio, which acquired Altor BioScience.
Approval was based on findings from the single arm, phase 2/3 open-label QUILT-3.032 study, which included 77 patients with BCG-unresponsive, high-risk disease following transurethral resection. All had Eastern Cooperative Oncology Group performance status of 0-2.
Patients received nogapendekin alfa inbakicept-pmln induction via intravesical instillation with BCG followed by maintenance therapy for up to 37 months.
According to the FDA’s press release, 62% of patients had a complete response, defined as a negative cystoscopy and urine cytology; 58% of those with a complete response had a duration of response lasting at least 12 months and 40% had a duration of response lasting 24 months or longer.
The safety of the combination was evaluated in a cohort of 88 patients. Serious adverse reactions occurred in 16% of patients. The most common treatment-emergent adverse effects included dysuria, pollakiuria, and hematuria, which are associated with intravesical BCG; 86% of these events were grade 1 or 2. Overall, 7% of patients discontinued the combination owing to adverse reactions.
The recommended dose is 400 mcg administered intravesically with BCG once a week for 6 weeks as induction therapy, with an option for a second induction course if patients don’t achieve a complete response at 3 months. The recommended maintenance therapy dose is 400 mcg with BCG once a week for 3 weeks at months 4, 7, 10, 13, and 19. Patients who achieve a complete response at 25 months and beyond may receive maintenance instillations with BCG once a week for 3 weeks at months 25, 31, and 37. The maximum treatment duration is 37 months.
The FDA recommends discontinuing treatment if disease persists after second induction or owing to disease recurrence, progression, or unacceptable toxicity.
A version of this article appeared on Medscape.com.
Specifically, the agent is approved to treat patients with BCG-unresponsive non–muscle-invasive bladder cancer carcinoma in situ with or without Ta or T1 papillary disease.
The FDA declined an initial approval for the combination in May 2023 because of deficiencies the agency observed during its prelicense inspection of third-party manufacturing organizations. In October 2023, ImmunityBio resubmitted the Biologics License Application, which was accepted.
The new therapy represents addresses “an unmet need” in this high-risk bladder cancer population, the company stated in a press release announcing the initial study findings. Typically, patients with intermediate or high-risk disease undergo bladder tumor resection followed by treatment with BCG, but the cancer recurs in up to 50% of patients, including those who experience a complete response, explained ImmunityBio, which acquired Altor BioScience.
Approval was based on findings from the single arm, phase 2/3 open-label QUILT-3.032 study, which included 77 patients with BCG-unresponsive, high-risk disease following transurethral resection. All had Eastern Cooperative Oncology Group performance status of 0-2.
Patients received nogapendekin alfa inbakicept-pmln induction via intravesical instillation with BCG followed by maintenance therapy for up to 37 months.
According to the FDA’s press release, 62% of patients had a complete response, defined as a negative cystoscopy and urine cytology; 58% of those with a complete response had a duration of response lasting at least 12 months and 40% had a duration of response lasting 24 months or longer.
The safety of the combination was evaluated in a cohort of 88 patients. Serious adverse reactions occurred in 16% of patients. The most common treatment-emergent adverse effects included dysuria, pollakiuria, and hematuria, which are associated with intravesical BCG; 86% of these events were grade 1 or 2. Overall, 7% of patients discontinued the combination owing to adverse reactions.
The recommended dose is 400 mcg administered intravesically with BCG once a week for 6 weeks as induction therapy, with an option for a second induction course if patients don’t achieve a complete response at 3 months. The recommended maintenance therapy dose is 400 mcg with BCG once a week for 3 weeks at months 4, 7, 10, 13, and 19. Patients who achieve a complete response at 25 months and beyond may receive maintenance instillations with BCG once a week for 3 weeks at months 25, 31, and 37. The maximum treatment duration is 37 months.
The FDA recommends discontinuing treatment if disease persists after second induction or owing to disease recurrence, progression, or unacceptable toxicity.
A version of this article appeared on Medscape.com.
FDA OKs Subcutaneous Vedolizumab for Crohn’s Maintenance Therapy
The move follows the FDA’s approval last year of subcutaneous vedolizumab for maintenance treatment of adults with moderately to severely active ulcerative colitis (UC).
The humanized immunoglobulin G1 monoclonal antibody is available as a single-dose prefilled pen (Entyvio Pen).
The FDA first approved the IV formulation of the biologic in 2014 for patients with moderate to severe UC and CD who cannot tolerate other therapies or in whom such therapies have failed.
The approval of subcutaneous vedolizumab for maintenance treatment of CD is based on the phase 3, randomized, double-blind, placebo-controlled VISIBLE 2 trial.
The trial enrolled 409 adult patients with moderately to severely active CD who had clinical response at week 6 following two doses of open-label IV vedolizumab at weeks 0 and 2.
At week 6, they were randomly allocated in a 2:1 ratio to receive vedolizumab 108 mg administered by subcutaneous injection or placebo every 2 weeks. The primary endpoint was clinical remission at week 52, which was defined as a total Crohn’s Disease Activity Index score ≤ 150.
The results showed that significantly more patients receiving subcutaneous vedolizumab than placebo achieved long-term clinical remission (48% vs 34%; P < .01), the company said in a news release.
The safety profile of subcutaneous vedolizumab is generally consistent with the known safety profile of IV vedolizumab, with the addition of injection-site reactions (including injection-site erythema, rash, pruritus, swelling, bruising, hematoma, pain, urticaria, and edema).
“Crohn’s disease is a complex and usually progressive disease for which an appropriate management plan is critical. My primary goal as a clinician is always to get patients to achieve remission,” Timothy Ritter, MD, senior medical director, GI Alliance Research, and assistant professor of medicine, Burnett School of Medicine at TCU, Fort Worth, Texas, said in the news release.
“In VISIBLE 2, about half of patients treated with Entyvio SC achieved long-term clinical remission. The data from VISIBLE 2 reaffirm the well-established efficacy profile of Entyvio, regardless of route of administration,” Dr. Ritter added.
A version of this article appeared on Medscape.com.
The move follows the FDA’s approval last year of subcutaneous vedolizumab for maintenance treatment of adults with moderately to severely active ulcerative colitis (UC).
The humanized immunoglobulin G1 monoclonal antibody is available as a single-dose prefilled pen (Entyvio Pen).
The FDA first approved the IV formulation of the biologic in 2014 for patients with moderate to severe UC and CD who cannot tolerate other therapies or in whom such therapies have failed.
The approval of subcutaneous vedolizumab for maintenance treatment of CD is based on the phase 3, randomized, double-blind, placebo-controlled VISIBLE 2 trial.
The trial enrolled 409 adult patients with moderately to severely active CD who had clinical response at week 6 following two doses of open-label IV vedolizumab at weeks 0 and 2.
At week 6, they were randomly allocated in a 2:1 ratio to receive vedolizumab 108 mg administered by subcutaneous injection or placebo every 2 weeks. The primary endpoint was clinical remission at week 52, which was defined as a total Crohn’s Disease Activity Index score ≤ 150.
The results showed that significantly more patients receiving subcutaneous vedolizumab than placebo achieved long-term clinical remission (48% vs 34%; P < .01), the company said in a news release.
The safety profile of subcutaneous vedolizumab is generally consistent with the known safety profile of IV vedolizumab, with the addition of injection-site reactions (including injection-site erythema, rash, pruritus, swelling, bruising, hematoma, pain, urticaria, and edema).
“Crohn’s disease is a complex and usually progressive disease for which an appropriate management plan is critical. My primary goal as a clinician is always to get patients to achieve remission,” Timothy Ritter, MD, senior medical director, GI Alliance Research, and assistant professor of medicine, Burnett School of Medicine at TCU, Fort Worth, Texas, said in the news release.
“In VISIBLE 2, about half of patients treated with Entyvio SC achieved long-term clinical remission. The data from VISIBLE 2 reaffirm the well-established efficacy profile of Entyvio, regardless of route of administration,” Dr. Ritter added.
A version of this article appeared on Medscape.com.
The move follows the FDA’s approval last year of subcutaneous vedolizumab for maintenance treatment of adults with moderately to severely active ulcerative colitis (UC).
The humanized immunoglobulin G1 monoclonal antibody is available as a single-dose prefilled pen (Entyvio Pen).
The FDA first approved the IV formulation of the biologic in 2014 for patients with moderate to severe UC and CD who cannot tolerate other therapies or in whom such therapies have failed.
The approval of subcutaneous vedolizumab for maintenance treatment of CD is based on the phase 3, randomized, double-blind, placebo-controlled VISIBLE 2 trial.
The trial enrolled 409 adult patients with moderately to severely active CD who had clinical response at week 6 following two doses of open-label IV vedolizumab at weeks 0 and 2.
At week 6, they were randomly allocated in a 2:1 ratio to receive vedolizumab 108 mg administered by subcutaneous injection or placebo every 2 weeks. The primary endpoint was clinical remission at week 52, which was defined as a total Crohn’s Disease Activity Index score ≤ 150.
The results showed that significantly more patients receiving subcutaneous vedolizumab than placebo achieved long-term clinical remission (48% vs 34%; P < .01), the company said in a news release.
The safety profile of subcutaneous vedolizumab is generally consistent with the known safety profile of IV vedolizumab, with the addition of injection-site reactions (including injection-site erythema, rash, pruritus, swelling, bruising, hematoma, pain, urticaria, and edema).
“Crohn’s disease is a complex and usually progressive disease for which an appropriate management plan is critical. My primary goal as a clinician is always to get patients to achieve remission,” Timothy Ritter, MD, senior medical director, GI Alliance Research, and assistant professor of medicine, Burnett School of Medicine at TCU, Fort Worth, Texas, said in the news release.
“In VISIBLE 2, about half of patients treated with Entyvio SC achieved long-term clinical remission. The data from VISIBLE 2 reaffirm the well-established efficacy profile of Entyvio, regardless of route of administration,” Dr. Ritter added.
A version of this article appeared on Medscape.com.
Second Ustekinumab Biosimilar Gets FDA Approval
The US Food and Drug Administration (FDA) has approved the biosimilar ustekinumab-aekn (Selarsdi) for the treatment of moderate to severe plaque psoriasis and psoriatic arthritis in adults and pediatric patients aged 6 years or older.
This is the second ustekinumab biosimilar approved by the regulatory agency and is the second biosimilar approval in the United States for the Icelandic pharmaceutical company Alvotech in partnership with Teva Pharmaceuticals.
Ustekinumab (Stelara) is a human monoclonal antibody targeting interleukin (IL)–12 and IL-23. The drug, manufactured by Johnson & Johnson, totaled nearly $7 billion in sales in 2023 alone, according a press release.
“Bringing Selarsdi to market in the US early next year presents a significant opportunity to improve patient access to a vital biologic in inflammatory disease and contribute to the reduction of inflationary pressure in healthcare costs,” the chairman and CEO of Alvotech said in the release.
The first ustekinumab biosimilar, ustekinumab-auub (Wezlana), was approved by the FDA in on October 31, 2023 and is interchangeable with the reference product. This allows pharmacists to substitute the biosimilar for the reference product without involving the prescribing clinician (according to state law). Besides psoriasis and psoriatic arthritis, ustekinumab-auub was also approved for treating moderate to severely active Crohn’s disease and ulcerative colitis. Ustekinumab-aekn does not have an interchangeability designation and was not approved for Crohn’s disease or ulcerative colitis.
The approval of ustekinumab-aekn was based on two clinical studies. A randomized, double blind, multicenter, 52-week study of 581 patients with moderate to severe plaque psoriasis demonstrated that the biosimilar was as effective as the reference product, with equivalent safety and immunogenicity profiles. A phase 1, randomized, double-blind, single-dose, parallel-group, three-arm study also compared the pharmacokinetic profile of the biosimilar to ustekinumab in 294 healthy adults.
Ustekinumab-aekn is expected to be marketed in the United States on or after February 21, 2025 per a settlement and license agreement with Johnson & Johnson.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved the biosimilar ustekinumab-aekn (Selarsdi) for the treatment of moderate to severe plaque psoriasis and psoriatic arthritis in adults and pediatric patients aged 6 years or older.
This is the second ustekinumab biosimilar approved by the regulatory agency and is the second biosimilar approval in the United States for the Icelandic pharmaceutical company Alvotech in partnership with Teva Pharmaceuticals.
Ustekinumab (Stelara) is a human monoclonal antibody targeting interleukin (IL)–12 and IL-23. The drug, manufactured by Johnson & Johnson, totaled nearly $7 billion in sales in 2023 alone, according a press release.
“Bringing Selarsdi to market in the US early next year presents a significant opportunity to improve patient access to a vital biologic in inflammatory disease and contribute to the reduction of inflationary pressure in healthcare costs,” the chairman and CEO of Alvotech said in the release.
The first ustekinumab biosimilar, ustekinumab-auub (Wezlana), was approved by the FDA in on October 31, 2023 and is interchangeable with the reference product. This allows pharmacists to substitute the biosimilar for the reference product without involving the prescribing clinician (according to state law). Besides psoriasis and psoriatic arthritis, ustekinumab-auub was also approved for treating moderate to severely active Crohn’s disease and ulcerative colitis. Ustekinumab-aekn does not have an interchangeability designation and was not approved for Crohn’s disease or ulcerative colitis.
The approval of ustekinumab-aekn was based on two clinical studies. A randomized, double blind, multicenter, 52-week study of 581 patients with moderate to severe plaque psoriasis demonstrated that the biosimilar was as effective as the reference product, with equivalent safety and immunogenicity profiles. A phase 1, randomized, double-blind, single-dose, parallel-group, three-arm study also compared the pharmacokinetic profile of the biosimilar to ustekinumab in 294 healthy adults.
Ustekinumab-aekn is expected to be marketed in the United States on or after February 21, 2025 per a settlement and license agreement with Johnson & Johnson.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved the biosimilar ustekinumab-aekn (Selarsdi) for the treatment of moderate to severe plaque psoriasis and psoriatic arthritis in adults and pediatric patients aged 6 years or older.
This is the second ustekinumab biosimilar approved by the regulatory agency and is the second biosimilar approval in the United States for the Icelandic pharmaceutical company Alvotech in partnership with Teva Pharmaceuticals.
Ustekinumab (Stelara) is a human monoclonal antibody targeting interleukin (IL)–12 and IL-23. The drug, manufactured by Johnson & Johnson, totaled nearly $7 billion in sales in 2023 alone, according a press release.
“Bringing Selarsdi to market in the US early next year presents a significant opportunity to improve patient access to a vital biologic in inflammatory disease and contribute to the reduction of inflationary pressure in healthcare costs,” the chairman and CEO of Alvotech said in the release.
The first ustekinumab biosimilar, ustekinumab-auub (Wezlana), was approved by the FDA in on October 31, 2023 and is interchangeable with the reference product. This allows pharmacists to substitute the biosimilar for the reference product without involving the prescribing clinician (according to state law). Besides psoriasis and psoriatic arthritis, ustekinumab-auub was also approved for treating moderate to severely active Crohn’s disease and ulcerative colitis. Ustekinumab-aekn does not have an interchangeability designation and was not approved for Crohn’s disease or ulcerative colitis.
The approval of ustekinumab-aekn was based on two clinical studies. A randomized, double blind, multicenter, 52-week study of 581 patients with moderate to severe plaque psoriasis demonstrated that the biosimilar was as effective as the reference product, with equivalent safety and immunogenicity profiles. A phase 1, randomized, double-blind, single-dose, parallel-group, three-arm study also compared the pharmacokinetic profile of the biosimilar to ustekinumab in 294 healthy adults.
Ustekinumab-aekn is expected to be marketed in the United States on or after February 21, 2025 per a settlement and license agreement with Johnson & Johnson.
A version of this article appeared on Medscape.com.
FDA Approves AI Diagnostic Tool for Early Sepsis Detection
The US Food and Drug Administration (FDA) has approved a medical device named the Sepsis ImmunoScore, which is an artificial intelligence/machine learning software, to guide rapid diagnosis and prediction of sepsis. The authorization was granted through the FDA’s De Novo pathway.
Sepsis is a complex condition, so diagnosing it early is difficult and has been a decades-long challenge for the US healthcare system.
Using both biomarkers and clinical data with the assistance of AI, the Sepsis ImmunoScore helps assess the risk for the presence of or progression to sepsis within 24 hours of patient evaluation in the emergency department or hospital. By considering 22 diverse parameters, the AI-powered tool provides a comprehensive evaluation of the patient’s biological condition, resulting in a risk score and categorization into four distinct risk levels.
It’s important to note that this system is not an alert mechanism. These risk categories are correlated with the risk for patient deterioration, including length of hospital stay, in-hospital mortality, and the need for escalated care within 24 hours (such as intensive care unit admission, mechanical ventilation, or vasopressor use). The diagnostic software is integrated directly into hospital electronic medical records.
This is the first AI diagnostic tool for sepsis to receive marketing authorization from the FDA.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved a medical device named the Sepsis ImmunoScore, which is an artificial intelligence/machine learning software, to guide rapid diagnosis and prediction of sepsis. The authorization was granted through the FDA’s De Novo pathway.
Sepsis is a complex condition, so diagnosing it early is difficult and has been a decades-long challenge for the US healthcare system.
Using both biomarkers and clinical data with the assistance of AI, the Sepsis ImmunoScore helps assess the risk for the presence of or progression to sepsis within 24 hours of patient evaluation in the emergency department or hospital. By considering 22 diverse parameters, the AI-powered tool provides a comprehensive evaluation of the patient’s biological condition, resulting in a risk score and categorization into four distinct risk levels.
It’s important to note that this system is not an alert mechanism. These risk categories are correlated with the risk for patient deterioration, including length of hospital stay, in-hospital mortality, and the need for escalated care within 24 hours (such as intensive care unit admission, mechanical ventilation, or vasopressor use). The diagnostic software is integrated directly into hospital electronic medical records.
This is the first AI diagnostic tool for sepsis to receive marketing authorization from the FDA.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved a medical device named the Sepsis ImmunoScore, which is an artificial intelligence/machine learning software, to guide rapid diagnosis and prediction of sepsis. The authorization was granted through the FDA’s De Novo pathway.
Sepsis is a complex condition, so diagnosing it early is difficult and has been a decades-long challenge for the US healthcare system.
Using both biomarkers and clinical data with the assistance of AI, the Sepsis ImmunoScore helps assess the risk for the presence of or progression to sepsis within 24 hours of patient evaluation in the emergency department or hospital. By considering 22 diverse parameters, the AI-powered tool provides a comprehensive evaluation of the patient’s biological condition, resulting in a risk score and categorization into four distinct risk levels.
It’s important to note that this system is not an alert mechanism. These risk categories are correlated with the risk for patient deterioration, including length of hospital stay, in-hospital mortality, and the need for escalated care within 24 hours (such as intensive care unit admission, mechanical ventilation, or vasopressor use). The diagnostic software is integrated directly into hospital electronic medical records.
This is the first AI diagnostic tool for sepsis to receive marketing authorization from the FDA.
A version of this article appeared on Medscape.com.
FDA Expands Enhertu Indication to HER2-Positive Solid Tumors
The agent had already been approved for several cancer types, including certain patients with unresectable or metastatic HER2-positive breast cancer as well as adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who had received a prior trastuzumab-based regimen.
The current accelerated approval is the first tumor-agnostic approval of a HER2-directed therapy and antibody drug conjugate.
“Until approval of trastuzumab deruxtecan, patients with metastatic HER2-positive tumors have had limited treatment options,” Funda Meric-Bernstam, MD, chair of investigational cancer therapeutics at the University of Texas MD Anderson Cancer Center, Houston, said in an AstraZeneca press statement. “Based on the clinically meaningful response rates across clinical trials, this tumor-agnostic approval means that patients may now be treated with a HER2-directed medicine.”
Approval was based on findings in 192 patients enrolled in either the DESTINY-PanTumor02 trial, the DESTINY-Lung01 trial, or the DESTINY-CRC02 trial. Patients in the multicenter trials underwent treatment until disease progression, death, withdrawal of consent or unacceptable toxicity.
Confirmed objective response rates were 51.4%, 52.9%, and 46.9% in the three studies, respectively. Median duration of response was 19.4, 6.9, and 5.5 months, respectively.
The most common adverse reactions occurring in at least 20% of patients included decreased white blood cell count, hemoglobin, lymphocyte count, and neutrophil count, as well as nausea, fatigue, platelet count, vomiting, alopecia, diarrhea, stomatitis, and upper respiratory tract infection.
Full prescribing information includes a boxed warning about the risk for interstitial lung disease and embryo-fetal toxicity.
The recommended dosage is 5.4 mg/kg given as an intravenous infusion one every 3 weeks until disease progression or unacceptable toxicity.
A version of this article appeared on Medscape.com.
The agent had already been approved for several cancer types, including certain patients with unresectable or metastatic HER2-positive breast cancer as well as adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who had received a prior trastuzumab-based regimen.
The current accelerated approval is the first tumor-agnostic approval of a HER2-directed therapy and antibody drug conjugate.
“Until approval of trastuzumab deruxtecan, patients with metastatic HER2-positive tumors have had limited treatment options,” Funda Meric-Bernstam, MD, chair of investigational cancer therapeutics at the University of Texas MD Anderson Cancer Center, Houston, said in an AstraZeneca press statement. “Based on the clinically meaningful response rates across clinical trials, this tumor-agnostic approval means that patients may now be treated with a HER2-directed medicine.”
Approval was based on findings in 192 patients enrolled in either the DESTINY-PanTumor02 trial, the DESTINY-Lung01 trial, or the DESTINY-CRC02 trial. Patients in the multicenter trials underwent treatment until disease progression, death, withdrawal of consent or unacceptable toxicity.
Confirmed objective response rates were 51.4%, 52.9%, and 46.9% in the three studies, respectively. Median duration of response was 19.4, 6.9, and 5.5 months, respectively.
The most common adverse reactions occurring in at least 20% of patients included decreased white blood cell count, hemoglobin, lymphocyte count, and neutrophil count, as well as nausea, fatigue, platelet count, vomiting, alopecia, diarrhea, stomatitis, and upper respiratory tract infection.
Full prescribing information includes a boxed warning about the risk for interstitial lung disease and embryo-fetal toxicity.
The recommended dosage is 5.4 mg/kg given as an intravenous infusion one every 3 weeks until disease progression or unacceptable toxicity.
A version of this article appeared on Medscape.com.
The agent had already been approved for several cancer types, including certain patients with unresectable or metastatic HER2-positive breast cancer as well as adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who had received a prior trastuzumab-based regimen.
The current accelerated approval is the first tumor-agnostic approval of a HER2-directed therapy and antibody drug conjugate.
“Until approval of trastuzumab deruxtecan, patients with metastatic HER2-positive tumors have had limited treatment options,” Funda Meric-Bernstam, MD, chair of investigational cancer therapeutics at the University of Texas MD Anderson Cancer Center, Houston, said in an AstraZeneca press statement. “Based on the clinically meaningful response rates across clinical trials, this tumor-agnostic approval means that patients may now be treated with a HER2-directed medicine.”
Approval was based on findings in 192 patients enrolled in either the DESTINY-PanTumor02 trial, the DESTINY-Lung01 trial, or the DESTINY-CRC02 trial. Patients in the multicenter trials underwent treatment until disease progression, death, withdrawal of consent or unacceptable toxicity.
Confirmed objective response rates were 51.4%, 52.9%, and 46.9% in the three studies, respectively. Median duration of response was 19.4, 6.9, and 5.5 months, respectively.
The most common adverse reactions occurring in at least 20% of patients included decreased white blood cell count, hemoglobin, lymphocyte count, and neutrophil count, as well as nausea, fatigue, platelet count, vomiting, alopecia, diarrhea, stomatitis, and upper respiratory tract infection.
Full prescribing information includes a boxed warning about the risk for interstitial lung disease and embryo-fetal toxicity.
The recommended dosage is 5.4 mg/kg given as an intravenous infusion one every 3 weeks until disease progression or unacceptable toxicity.
A version of this article appeared on Medscape.com.
FDA OKs First-in-Class Agent for Pulmonary Arterial Hypertension
The US Food and Drug Administration (FDA) has approved sotatercept (Winrevair, Merck), for the treatment of adults with pulmonary arterial hypertension (PAH), World Health Organization (WHO) Group 1, to increase exercise capacity, improve WHO functional class, and reduce the risk for clinical worsening events.
“Sotatercept added to background therapy has the potential to become a new standard-of-care option for patients with pulmonary arterial hypertension,” added coinvestigator Aaron B. Waxman, MD, PhD, executive director of the Center for Pulmonary Heart Diseases at Brigham and Women’s Hospital, Boston.
The approval was based on results of the phase 3 STELLAR study, a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which, 323 patients with PAH (WHO Group 1, functional class II or III) were randomly assigned 1:1 to add sotatercept or placebo to stable background therapy.
The results showed that sotatercept, administered subcutaneously every 3 weeks for 24 weeks, improved average 6-minute walk distance from baseline by a significant and clinically meaningful 40.8 meters compared with placebo for the trial’s primary efficacy endpoint (P < .001).
Sotatercept also led to significant improvement in multiple secondary outcome measures, including:
- Reduction in the risk for death from any cause or PAH clinical worsening events by 84% vs background therapy alone (number of events: 9 vs 42; hazard ratio [HR], 0.16; P < .001)
- Improvement in FC from baseline at 24 weeks in 29% of patients compared with 14% of patients treated with placebo (P < .001)
- Improvement in pulmonary vascular resistance (PVR), with an average 235 dyn/sec/cm5 reduction in PVR from baseline (P < .001)
- Improvement from baseline in N-terminal pro–B-type natriuretic peptide (NT-proBNP) levels. The median treatment difference in NT-proBNP between sotatercept and placebo was -442 pg/mL (P < .001)
The results were reported last year at the joint scientific sessions of the American College of Cardiology and the World Heart Federation, with simultaneous publication in The New England Journal of Medicine.
Sotatercept injection may be administered by patients or caregivers with guidance, training, and follow-up from a healthcare provider. The recommended starting dose is 0.3 mg/kg. The recommended target dose is 0.7 mg/kg every 3 weeks.
Sotatercept may increase hemoglobin, may lead to erythrocytosis, and may decrease platelet count and lead to severe thrombocytopenia. Treatment should not be initiated if platelet count is < 50,000/mm3.
Hemoglobin and platelets should be monitored before each dose of sotatercept for the first five doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required.
Full prescribing information is available online.
Merck estimates that sotatercept will be available for dispensing by select specialty pharmacies in the United States by the end of April 2024.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved sotatercept (Winrevair, Merck), for the treatment of adults with pulmonary arterial hypertension (PAH), World Health Organization (WHO) Group 1, to increase exercise capacity, improve WHO functional class, and reduce the risk for clinical worsening events.
“Sotatercept added to background therapy has the potential to become a new standard-of-care option for patients with pulmonary arterial hypertension,” added coinvestigator Aaron B. Waxman, MD, PhD, executive director of the Center for Pulmonary Heart Diseases at Brigham and Women’s Hospital, Boston.
The approval was based on results of the phase 3 STELLAR study, a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which, 323 patients with PAH (WHO Group 1, functional class II or III) were randomly assigned 1:1 to add sotatercept or placebo to stable background therapy.
The results showed that sotatercept, administered subcutaneously every 3 weeks for 24 weeks, improved average 6-minute walk distance from baseline by a significant and clinically meaningful 40.8 meters compared with placebo for the trial’s primary efficacy endpoint (P < .001).
Sotatercept also led to significant improvement in multiple secondary outcome measures, including:
- Reduction in the risk for death from any cause or PAH clinical worsening events by 84% vs background therapy alone (number of events: 9 vs 42; hazard ratio [HR], 0.16; P < .001)
- Improvement in FC from baseline at 24 weeks in 29% of patients compared with 14% of patients treated with placebo (P < .001)
- Improvement in pulmonary vascular resistance (PVR), with an average 235 dyn/sec/cm5 reduction in PVR from baseline (P < .001)
- Improvement from baseline in N-terminal pro–B-type natriuretic peptide (NT-proBNP) levels. The median treatment difference in NT-proBNP between sotatercept and placebo was -442 pg/mL (P < .001)
The results were reported last year at the joint scientific sessions of the American College of Cardiology and the World Heart Federation, with simultaneous publication in The New England Journal of Medicine.
Sotatercept injection may be administered by patients or caregivers with guidance, training, and follow-up from a healthcare provider. The recommended starting dose is 0.3 mg/kg. The recommended target dose is 0.7 mg/kg every 3 weeks.
Sotatercept may increase hemoglobin, may lead to erythrocytosis, and may decrease platelet count and lead to severe thrombocytopenia. Treatment should not be initiated if platelet count is < 50,000/mm3.
Hemoglobin and platelets should be monitored before each dose of sotatercept for the first five doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required.
Full prescribing information is available online.
Merck estimates that sotatercept will be available for dispensing by select specialty pharmacies in the United States by the end of April 2024.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved sotatercept (Winrevair, Merck), for the treatment of adults with pulmonary arterial hypertension (PAH), World Health Organization (WHO) Group 1, to increase exercise capacity, improve WHO functional class, and reduce the risk for clinical worsening events.
“Sotatercept added to background therapy has the potential to become a new standard-of-care option for patients with pulmonary arterial hypertension,” added coinvestigator Aaron B. Waxman, MD, PhD, executive director of the Center for Pulmonary Heart Diseases at Brigham and Women’s Hospital, Boston.
The approval was based on results of the phase 3 STELLAR study, a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which, 323 patients with PAH (WHO Group 1, functional class II or III) were randomly assigned 1:1 to add sotatercept or placebo to stable background therapy.
The results showed that sotatercept, administered subcutaneously every 3 weeks for 24 weeks, improved average 6-minute walk distance from baseline by a significant and clinically meaningful 40.8 meters compared with placebo for the trial’s primary efficacy endpoint (P < .001).
Sotatercept also led to significant improvement in multiple secondary outcome measures, including:
- Reduction in the risk for death from any cause or PAH clinical worsening events by 84% vs background therapy alone (number of events: 9 vs 42; hazard ratio [HR], 0.16; P < .001)
- Improvement in FC from baseline at 24 weeks in 29% of patients compared with 14% of patients treated with placebo (P < .001)
- Improvement in pulmonary vascular resistance (PVR), with an average 235 dyn/sec/cm5 reduction in PVR from baseline (P < .001)
- Improvement from baseline in N-terminal pro–B-type natriuretic peptide (NT-proBNP) levels. The median treatment difference in NT-proBNP between sotatercept and placebo was -442 pg/mL (P < .001)
The results were reported last year at the joint scientific sessions of the American College of Cardiology and the World Heart Federation, with simultaneous publication in The New England Journal of Medicine.
Sotatercept injection may be administered by patients or caregivers with guidance, training, and follow-up from a healthcare provider. The recommended starting dose is 0.3 mg/kg. The recommended target dose is 0.7 mg/kg every 3 weeks.
Sotatercept may increase hemoglobin, may lead to erythrocytosis, and may decrease platelet count and lead to severe thrombocytopenia. Treatment should not be initiated if platelet count is < 50,000/mm3.
Hemoglobin and platelets should be monitored before each dose of sotatercept for the first five doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required.
Full prescribing information is available online.
Merck estimates that sotatercept will be available for dispensing by select specialty pharmacies in the United States by the end of April 2024.
A version of this article appeared on Medscape.com.
Measles Control So Far in 2024: ‘Not Off to a Great Start’
Just over 2 months into 2024, measles cases in the United States aren’t looking great.
The recent rise in cases across the U.S. is linked to unvaccinated travelers, lower than ideal vaccination rates, and misinformation, experts said.
The Centers for Disease Control and Prevention has identified 45 cases of measles in 17 jurisdictions across the U.S. As of March 7, the federal health agency reported measles cases in Arizona, California, Florida, Georgia, Illinois, Indiana, Louisiana, Maryland, Michigan, Minnesota, Missouri, New Jersey, New York City, Ohio, Pennsylvania, Virginia, and Washington.
As for the 45 cases, “that’s almost as many as we had for the entire calendar year of 2023,” said Sarah Lim, MD, a medical specialist at the Minnesota Department of Health. “So we’re really not off to a great start.” (For context, there were 58 officially reported measles cases last year.)
Chicago is having a measles outbreak — with eight cases reported so far. All but one case has been linked to a migrant child at a city shelter. Given the potential for rapid spread — measles is relatively rare here but potentially very serious — the CDC sent a team of experts to investigate and to help keep this outbreak from growing further.
Sometimes Deadly
About 30% of children have measles symptoms and about 25% end up hospitalized. Complications include diarrhea, a whole-body rash, ear infections that can lead to permanent deafness, and pneumonia. Pneumonia with measles can be so serious that 1 in 20 affected children die. Measles can also cause inflammation of the brain called encephalitis in about 1 in 1,000 children, sometimes causing epilepsy or permanent brain damage.
As with long COVID, some effects can last beyond the early infection. For example, measles “can wipe out immune memory that protects you against other bacterial and viral pathogens,” Dr. Lim said at a media briefing sponsored by the Infectious Diseases Society of America. This vulnerability to other infections can last up to 3 years after the early infection, she noted.
Overall, measles kills between 1 and 3 people infected per thousand, mostly children.
Vaccine Misinformation Playing a Role
Vaccine misinformation is partly behind the uptick, and while many cases are mild, “this can be a devastating disease,” said Joshua Barocas, MD, associate professor of medicine in the divisions of General Internal Medicine and Infectious Diseases at the University of Colorado School of Medicine.
“I’m a parent myself. Parents are flooded with tons of information, some of that time being misinformation,” he said at the media briefing. “If you are a parent who’s been on the fence [about vaccination], now is the time, given the outbreak potential and the outbreaks that we’re seeing.”
Vaccine misinformation “is about as old as vaccines themselves,” Dr. Lim said. Concerns about the MMR vaccine, which includes measles protection, are not new.
“It does seem to change periodically — new things bubble up, new ideas bubble up, and the problem is that it is like the old saying that ‘a lie can get halfway around the world before the truth can get its boots on.’ ” Social media helps to amplify vaccine misinformation, she said.
“You don’t want to scare people unnecessarily — but reminding people what these childhood diseases really look like and what they do is incredibly important,” Dr. Lim said. “It’s so much easier to see stories about potential side effects of vaccines than it is to see stories about parents whose children were in intensive care for 2 weeks with pneumonia because of a severe case of measles.”
Dr. Barocas said misinformation is sometimes deliberate, sometimes not. Regardless, “our job as infectious disease physicians and public health professionals is not necessarily to put the counternarrative out there, but to continue to advocate for what we know works based on the best science and the best evidence.”
“And there is no reason to believe that vaccines are anything but helpful when it comes to preventing measles,” he noted.
Lifelong Protection in Most Cases
The MMR vaccine, typically given as two doses in childhood, offers 93% and then 97% protection against the highly contagious virus. During the 2022-to-2023 school year, the measles vaccination rate among kindergarten children nationwide was 92%. That sounds like a high rate, Dr. Lim said, “but because measles is so contagious, vaccination rates need to be 95% or higher to contain transmission.”
One person with measles can infect anywhere from 12 to 18 other people, she said. When an infected person coughs or sneezes, tiny droplets spread through the air. “And if someone is unvaccinated and exposed, 9 times out of 10, that person will go on to develop the disease.” She said given the high transmission rate, measles often spreads within families to infect multiple children.
If you know you’re not vaccinated but exposed, the advice is to get the measles shot as quickly as possible. “There is a recommendation to receive the MMR vaccine within 72 hours as post-exposure prophylaxis,” Dr. Lim said. “That’s a tight time window, but if you can do that, it reduces the risk of developing measles significantly.”
If you’re unsure or do not remember getting vaccinated against measles as a young child, your health care provider may be able to search state registries for an answer. If that doesn’t work, getting revaccinated with the MMR vaccine as an adult is an option. “There is no shame in getting caught up now,” Dr. Barocas said.
Dr. Lim agreed. “There is really no downside to getting additional doses.”
A version of this article appeared on WebMD.com.
Just over 2 months into 2024, measles cases in the United States aren’t looking great.
The recent rise in cases across the U.S. is linked to unvaccinated travelers, lower than ideal vaccination rates, and misinformation, experts said.
The Centers for Disease Control and Prevention has identified 45 cases of measles in 17 jurisdictions across the U.S. As of March 7, the federal health agency reported measles cases in Arizona, California, Florida, Georgia, Illinois, Indiana, Louisiana, Maryland, Michigan, Minnesota, Missouri, New Jersey, New York City, Ohio, Pennsylvania, Virginia, and Washington.
As for the 45 cases, “that’s almost as many as we had for the entire calendar year of 2023,” said Sarah Lim, MD, a medical specialist at the Minnesota Department of Health. “So we’re really not off to a great start.” (For context, there were 58 officially reported measles cases last year.)
Chicago is having a measles outbreak — with eight cases reported so far. All but one case has been linked to a migrant child at a city shelter. Given the potential for rapid spread — measles is relatively rare here but potentially very serious — the CDC sent a team of experts to investigate and to help keep this outbreak from growing further.
Sometimes Deadly
About 30% of children have measles symptoms and about 25% end up hospitalized. Complications include diarrhea, a whole-body rash, ear infections that can lead to permanent deafness, and pneumonia. Pneumonia with measles can be so serious that 1 in 20 affected children die. Measles can also cause inflammation of the brain called encephalitis in about 1 in 1,000 children, sometimes causing epilepsy or permanent brain damage.
As with long COVID, some effects can last beyond the early infection. For example, measles “can wipe out immune memory that protects you against other bacterial and viral pathogens,” Dr. Lim said at a media briefing sponsored by the Infectious Diseases Society of America. This vulnerability to other infections can last up to 3 years after the early infection, she noted.
Overall, measles kills between 1 and 3 people infected per thousand, mostly children.
Vaccine Misinformation Playing a Role
Vaccine misinformation is partly behind the uptick, and while many cases are mild, “this can be a devastating disease,” said Joshua Barocas, MD, associate professor of medicine in the divisions of General Internal Medicine and Infectious Diseases at the University of Colorado School of Medicine.
“I’m a parent myself. Parents are flooded with tons of information, some of that time being misinformation,” he said at the media briefing. “If you are a parent who’s been on the fence [about vaccination], now is the time, given the outbreak potential and the outbreaks that we’re seeing.”
Vaccine misinformation “is about as old as vaccines themselves,” Dr. Lim said. Concerns about the MMR vaccine, which includes measles protection, are not new.
“It does seem to change periodically — new things bubble up, new ideas bubble up, and the problem is that it is like the old saying that ‘a lie can get halfway around the world before the truth can get its boots on.’ ” Social media helps to amplify vaccine misinformation, she said.
“You don’t want to scare people unnecessarily — but reminding people what these childhood diseases really look like and what they do is incredibly important,” Dr. Lim said. “It’s so much easier to see stories about potential side effects of vaccines than it is to see stories about parents whose children were in intensive care for 2 weeks with pneumonia because of a severe case of measles.”
Dr. Barocas said misinformation is sometimes deliberate, sometimes not. Regardless, “our job as infectious disease physicians and public health professionals is not necessarily to put the counternarrative out there, but to continue to advocate for what we know works based on the best science and the best evidence.”
“And there is no reason to believe that vaccines are anything but helpful when it comes to preventing measles,” he noted.
Lifelong Protection in Most Cases
The MMR vaccine, typically given as two doses in childhood, offers 93% and then 97% protection against the highly contagious virus. During the 2022-to-2023 school year, the measles vaccination rate among kindergarten children nationwide was 92%. That sounds like a high rate, Dr. Lim said, “but because measles is so contagious, vaccination rates need to be 95% or higher to contain transmission.”
One person with measles can infect anywhere from 12 to 18 other people, she said. When an infected person coughs or sneezes, tiny droplets spread through the air. “And if someone is unvaccinated and exposed, 9 times out of 10, that person will go on to develop the disease.” She said given the high transmission rate, measles often spreads within families to infect multiple children.
If you know you’re not vaccinated but exposed, the advice is to get the measles shot as quickly as possible. “There is a recommendation to receive the MMR vaccine within 72 hours as post-exposure prophylaxis,” Dr. Lim said. “That’s a tight time window, but if you can do that, it reduces the risk of developing measles significantly.”
If you’re unsure or do not remember getting vaccinated against measles as a young child, your health care provider may be able to search state registries for an answer. If that doesn’t work, getting revaccinated with the MMR vaccine as an adult is an option. “There is no shame in getting caught up now,” Dr. Barocas said.
Dr. Lim agreed. “There is really no downside to getting additional doses.”
A version of this article appeared on WebMD.com.
Just over 2 months into 2024, measles cases in the United States aren’t looking great.
The recent rise in cases across the U.S. is linked to unvaccinated travelers, lower than ideal vaccination rates, and misinformation, experts said.
The Centers for Disease Control and Prevention has identified 45 cases of measles in 17 jurisdictions across the U.S. As of March 7, the federal health agency reported measles cases in Arizona, California, Florida, Georgia, Illinois, Indiana, Louisiana, Maryland, Michigan, Minnesota, Missouri, New Jersey, New York City, Ohio, Pennsylvania, Virginia, and Washington.
As for the 45 cases, “that’s almost as many as we had for the entire calendar year of 2023,” said Sarah Lim, MD, a medical specialist at the Minnesota Department of Health. “So we’re really not off to a great start.” (For context, there were 58 officially reported measles cases last year.)
Chicago is having a measles outbreak — with eight cases reported so far. All but one case has been linked to a migrant child at a city shelter. Given the potential for rapid spread — measles is relatively rare here but potentially very serious — the CDC sent a team of experts to investigate and to help keep this outbreak from growing further.
Sometimes Deadly
About 30% of children have measles symptoms and about 25% end up hospitalized. Complications include diarrhea, a whole-body rash, ear infections that can lead to permanent deafness, and pneumonia. Pneumonia with measles can be so serious that 1 in 20 affected children die. Measles can also cause inflammation of the brain called encephalitis in about 1 in 1,000 children, sometimes causing epilepsy or permanent brain damage.
As with long COVID, some effects can last beyond the early infection. For example, measles “can wipe out immune memory that protects you against other bacterial and viral pathogens,” Dr. Lim said at a media briefing sponsored by the Infectious Diseases Society of America. This vulnerability to other infections can last up to 3 years after the early infection, she noted.
Overall, measles kills between 1 and 3 people infected per thousand, mostly children.
Vaccine Misinformation Playing a Role
Vaccine misinformation is partly behind the uptick, and while many cases are mild, “this can be a devastating disease,” said Joshua Barocas, MD, associate professor of medicine in the divisions of General Internal Medicine and Infectious Diseases at the University of Colorado School of Medicine.
“I’m a parent myself. Parents are flooded with tons of information, some of that time being misinformation,” he said at the media briefing. “If you are a parent who’s been on the fence [about vaccination], now is the time, given the outbreak potential and the outbreaks that we’re seeing.”
Vaccine misinformation “is about as old as vaccines themselves,” Dr. Lim said. Concerns about the MMR vaccine, which includes measles protection, are not new.
“It does seem to change periodically — new things bubble up, new ideas bubble up, and the problem is that it is like the old saying that ‘a lie can get halfway around the world before the truth can get its boots on.’ ” Social media helps to amplify vaccine misinformation, she said.
“You don’t want to scare people unnecessarily — but reminding people what these childhood diseases really look like and what they do is incredibly important,” Dr. Lim said. “It’s so much easier to see stories about potential side effects of vaccines than it is to see stories about parents whose children were in intensive care for 2 weeks with pneumonia because of a severe case of measles.”
Dr. Barocas said misinformation is sometimes deliberate, sometimes not. Regardless, “our job as infectious disease physicians and public health professionals is not necessarily to put the counternarrative out there, but to continue to advocate for what we know works based on the best science and the best evidence.”
“And there is no reason to believe that vaccines are anything but helpful when it comes to preventing measles,” he noted.
Lifelong Protection in Most Cases
The MMR vaccine, typically given as two doses in childhood, offers 93% and then 97% protection against the highly contagious virus. During the 2022-to-2023 school year, the measles vaccination rate among kindergarten children nationwide was 92%. That sounds like a high rate, Dr. Lim said, “but because measles is so contagious, vaccination rates need to be 95% or higher to contain transmission.”
One person with measles can infect anywhere from 12 to 18 other people, she said. When an infected person coughs or sneezes, tiny droplets spread through the air. “And if someone is unvaccinated and exposed, 9 times out of 10, that person will go on to develop the disease.” She said given the high transmission rate, measles often spreads within families to infect multiple children.
If you know you’re not vaccinated but exposed, the advice is to get the measles shot as quickly as possible. “There is a recommendation to receive the MMR vaccine within 72 hours as post-exposure prophylaxis,” Dr. Lim said. “That’s a tight time window, but if you can do that, it reduces the risk of developing measles significantly.”
If you’re unsure or do not remember getting vaccinated against measles as a young child, your health care provider may be able to search state registries for an answer. If that doesn’t work, getting revaccinated with the MMR vaccine as an adult is an option. “There is no shame in getting caught up now,” Dr. Barocas said.
Dr. Lim agreed. “There is really no downside to getting additional doses.”
A version of this article appeared on WebMD.com.