As reported on Drugs, Scholar Rock says the US Food and Drug Administration’s review of its biologics license application for apitegromab, an investigational treatment for spinal muscular atrophy (SMA), remains on course for a potential decision by September 30, 2026.
The company will proceed with a single fill-finish manufacturing site after learning that the FDA classified its April 2026 inspection of Catalent Indiana, a Novo Nordisk company, as Official Action Indicated (OAI). Scholar Rock said it is working with the agency and plans to remove the Catalent Indiana facility from the application.
Second Facility Under FDA Review
Apitegromab’s March 2026 submission included two fill-finish sites, providing alternative manufacturing pathways during the regulatory review. The remaining US-based facility is now the sole site supporting the application.
According to Scholar Rock, the company and FDA agreed on the information needed to assess this second facility during a Type C meeting held in March. The company said it submitted the requested package ahead of schedule and that the agency’s assessment is ongoing.
Scholar Rock reported that commercial drug-product vials from the second facility have already been produced. The vials are being held by a third-party provider for final packaging and labeling should the therapy receive approval. The company characterized the site as compliant with FDA and European Medicines Agency expectations and noted that it has recently completed successful inspections by both regulators.
Potential New Option for SMA
Apitegromab is a fully human monoclonal antibody designed to inhibit activation of myostatin, a protein that limits skeletal-muscle growth. By binding precursor and inactive forms of myostatin within muscle tissue, the therapy is intended to improve motor function in children and adults with SMA.
The candidate has demonstrated positive results in a pivotal phase 3 trial, according to Scholar Rock, and the company describes it as the first SMA treatment candidate specifically aimed at skeletal muscle. Existing SMA therapies generally address the underlying genetic pathway or support production of survival motor neuron protein.
The FDA has granted the program Fast Track, Orphan Drug, and Rare Pediatric Disease designations. In Europe, apitegromab has received PRIME and orphan-medicinal-product status.
European Filing Also Being Updated
Scholar Rock is also discussing how to incorporate the second manufacturing location into its European marketing authorization application. The company said it will provide an update on the expected timing for a Committee for Medicinal Products for Human Use opinion once it reaches agreement with the EMA.
Apitegromab has not yet been cleared by the FDA, EMA, or any other regulator. While the manufacturing-site adjustment introduces an operational change to the filing, Scholar Rock maintains that it has sufficient product supply and is prepared to begin a US launch immediately if approval is granted by the September PDUFA deadline.
