Drugs

FDA Issues Complete Response Letter for Apitegromab

FDA has issued Scholar Rock a complete response letter for its BLA seeking approval of apitegromab, a first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA). The letter cited observations made during an FDA inspection of Catalent Indiana, a third-party fill-finish facility acquired by Novo Nordisk in late 2024, according to Scholar Rock. The issues were described as general site deficiencies and not specific to apitegromab’s efficacy, safety, or the manufacturing of its drug substance, it adds.

Catalent Indiana submitted a corrective action plan to FDA in August, and remediation efforts remain ongoing. Scholar Rock says it will resubmit the BLA once the facility’s compliance issues are resolved.

Apitegromab is described by the company as a monoclonal antibody that blocks myostatin activation in skeletal muscle. SMA is a rare genetic neuromuscular disorder marked by progressive muscle weakness and motor function decline. Existing treatments largely focus on preserving motor neurons but do not directly address muscle deterioration, according to the company.

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