FDA Roundup: Hemophilia B Therapy, Stelara Biosimilar Approval

FDA Approves First Weekly Subcutaneous Therapy for Hemophilia B

FDA Roundup: Hemophilia B Therapy, Stelara Biosimilar Approval / Tada Images - stock.adobe.com

The FDA has approved marstacimab-hncq (Hypmavzi) as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 year of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. The medication is administered weekly using an autoinjector pen and is the first subcutaneous option for patients with hemophilia B.

Marstacimab-hncq's approval is based on the results of the phase 3 BASIS trial, a large-scale clinical study involving 116 male patients with severe hemophilia A or B. Trial results, published in the journal Blood, demonstrated the effectiveness of marstacimab-hncq in preventing bleeding episodes.

Marstacimab-hncq is a novel treatment for hemophilia B. Unlike traditional therapies that replace clotting factors, it works by reducing the amount of anticoagulation proteins called tissue factor inhibitors. This increases the production of thrombin, an enzyme essential for blood clotting, which is expected to reduce or prevent bleeding episodes.

FDA Approves Imuldosa, Biosimilar to Stelara

The FDA has approved Dong-A ST’s ustekinumab-srlf (Imuldosa), a biosimilar to Stelara, for the treatment of patients with autoimmune diseases such as plaque psoriasis, psoriatic arthritis, Crohn’s disease and ulcerative colitis.

The approval is supported by results from phase 3 multi-regional clinical trials conducted in patients with plaque psoriasis. The primary endpoint was the change in the Psoriasis Area and Severity Index for skin symptoms. Clinical data showed that ustekinumab-srlf and its reference product, ustekinumab, were highly similar and had no clinically significant differences in quality, safety, or efficacy.

FDA Accepts Bayer’s NDA for Elinzanetant to Treat Menopause Symptoms

The FDA has accepted Bayer’s New Drug Application (NDA) for elinzanetant, an investigational non-hormonal treatment for moderate-to-severe vasomotor symptoms (VMS) associated with menopause. The therapy is the first dual neurokinin-1 and 3 receptor antagonist in late-stage clinical development and is administered orally once daily.

The NDA application is supported by positive results from the OASIS 1, 2 and 3 phase three studies evaluating the efficacy and safety of elinzanetant versus placebo. Findings from OASIS 1 and 2 were published late this summer in JAMA, and results of the phase three OASIS 3 study supporting efficacy and additional long-term safety data were presented at The Menopause Society annual meeting in September 2024.

FDA Grants Marketing Authorization to First OTC Flu, COVID-19 Combination Test

The FDA has granted marketing authorization to the Healgen Rapid Check COVID-19/Flu A&B Antigen Test for use by individuals experiencing respiratory symptoms. The over-the-counter (OTC) test is the first to be granted marketing authorization using a traditional premarket review pathway, as other similar tests are only currently available under emergency use authorization (EUA).

The granting of the De Novo marketing authorization was based on data from a study assessing the Healgen rapid test’s quality, accuracy and reliability in individuals with signs and symptoms of COVID-19 and influenza. The study found the test correctly identified 99% of negative and 92% of positive SARS-CoV-2 samples. It also correctly identified 99.9% of negative flu A and B samples, 92.5% of positive flu A samples and 90.5% of positive flu B samples.

The Healgen Rapid Check COVID-19/Flu A&B Antigen Test is authorized for use in individuals aged 14 years or older, or adult-collected samples from individuals aged 2 years or older, who are experiencing respiratory symptoms. The test, meant to be used within the first 5 days from when symptoms begin, uses a nasal swab to collect samples and delivers results in 15 minutes by detecting proteins from SARS-CoV-2 and influenza A and B.

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