FDA Policy/General

Scholar Rock Refiles BLA for Spinal Muscular Atrophy Therapy

Scholar Rock has resubmitted its BLA for apitegromab, an investigational therapy for children and adults with Spinal Muscular Atrophy (SMA), following alignment with FDA on manufacturing and supply issues that previously delayed approval. The resubmission follows a 9/2025 complete response letter that cited inspection findings at a contract manufacturing site operated by Catalent Indiana, now part of Novo Nordisk. FDA did not raise concerns about the drug’s clinical efficacy or safety at the time, according to Scholar Rock.

The company says subsequent discussions with the agency — including a Type A meeting in late 2025 and additional interactions in early 2026 — cleared the way for the company to refile its application ahead of a reinspection of the Indiana facility. According to the company, the FDA did not request further corrective actions following a recent site visit tied to remediation efforts.

As part of the updated submission, Scholar Rock says it added a second U.S.-based fill-finish manufacturing facility, a move intended to strengthen supply chain resilience and support anticipated global demand. The inclusion followed a 3/3 Type C meeting with FDA, and it agreed to the approach.

The company expects the agency to accept the application for review and set a user fee review action date for late September.

Apitegromab is designed to inhibit myostatin activation, targeting muscle growth pathways rather than the underlying genetic cause of SMA, according to the company. In a pivotal Phase 3 trial, the therapy demonstrated statistically significant and clinically meaningful improvements in motor function in patients already receiving SMN-targeted treatments, positioning it as a potential add-on therapy.

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