Biologics

Vyvgart sBLA Seeks Expanded Use

FDA has accepted for priority review an Argenx supplemental BLA seeking to expand the use of its myasthenia gravis drug Vyvgart (efgartigimod alfa-fcab) to adults with acetylcholine receptor antibody–negative generalized myasthenia gravis. The agency set a user fee review action target date of 5/10. If approved, the company says it would mark the first U.S. authorization of a targeted therapy for patients with seronegative generalized myasthenia gravis, a population that has historically faced limited treatment options.

The supplemental filing covers intravenous Vyvgart for adults with AChR antibody–seronegative disease, including patients who are MuSK-positive, LRP4-positive, or triple seronegative, according to the company. Vyvgart is already approved in the U.S. for adults with AChR antibody–positive generalized myasthenia gravis.

Argenx says the application is supported by data from the Phase 3 ADAPT SERON study, which met its primary endpoint by demonstrating a statistically significant improvement in disease-related daily functioning compared with placebo after four weeks of treatment. In the trial, patients treated with VYVGART achieved a mean 3.35-point improvement from baseline in the Myasthenia Gravis Activities of Daily Living score at week four, a change the company describes as clinically meaningful. Improvements were observed across multiple treatment cycles and across all seronegative subgroups, it adds.

Generalized myasthenia gravis is a rare autoimmune neuromuscular disease characterized by fluctuating muscle weakness that can be debilitating or life-threatening. About 20% of patients do not have detectable antibodies against the acetylcholine receptor and are classified as seronegative, with some harboring antibodies against other neuromuscular junction proteins and others lacking identifiable autoantibodies altogether, the company says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome