Drugs

Regeneron to Seek FDA OK for Myasthenia Gravis Drug

Regeneron Pharmaceuticals says it will seek FDA approval early next year for its investigational RNA-based therapy cemdisiran after announcing results from the Phase 3 NIMBLE trial where all primary and key secondary endpoints were met for treating generalized myasthenia gravis. Cemdisiran is described by the company as an siRNA that reduces circulating levels of complement factor 5 (C5). As a monotherapy in the trial, the therapy was associated with an average of 74% inhibition of complement activity.

The trial also assessed a combination of cemdisiran and pozelimab, a C5 antibody, according to Regeneron. The combination resulted in nearly 99% inhibition of complement activity, and it also met the primary and key secondary endpoints, though cemdisiran monotherapy was numerically better across these endpoints, it adds.

No patients discontinued cemdisiran due to side effects during the placebo-controlled portion of the study, according to the company. Adverse events were generally manageable, with the most common being mild infections and headaches, it says, adding that serious side effects occurred in 3% of patients on cemdisiran, compared with 9% on the combination and 14% on placebo. No meningococcal infections were reported.

Generalized myasthenia gravis is a rare autoimmune disease that affects about 85,000 people in the U.S., the company says. It causes muscle weakness that can impair vision, speech, swallowing, and mobility. Current treatments often require frequent dosing and may not provide sustained symptom control.

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