Scholar Rock: FDA Turns Away Apitegromab Application — Update

By Colin Kellaher

Scholar Rock Holding said the Food and Drug Administration has turned away its application seeking approval of apitegromab for the rare genetic neuromuscular disease spinal muscular atrophy due to issues at a contract manufacturer, sending shares of the biopharmaceutical company lower in premarket trading.

Scholar Rock on Tuesday said the FDA has issued a so-called complete response letter, indicating the agency won't approve the Cambridge, Mass., company's application in its current form.

Shares of Scholar Rock were recently down 14% to $27.95 in premarket trading.

Scholar Rock said the FDA cited observations identified during a routine general site inspection of Catalent Indiana, a third-party fill-finish plant acquired by Novo Nordisk last year.

The company said the FDA didn't raise any other approvability concerns, adding that it plans to resubmit its application upon Catalent Indiana's successful remediation of the agency's observations.

Scholar Rock said Catalent Indiana submitted a response in early August to address the FDA's observations and has been working rapidly to take corrective action.

The biotechnology company last month said issues at the Catalent Indiana facility had led to FDA setbacks for applications related to expanded approval of its Eylea HD eye drug and approval of a proposed lymphoma drug.

Scholar Rock previously said it was planning for a U.S. commercial launch of apitegromab this year based on an FDA target action date of Sept. 22.

The company has said an FDA green light would make apitegromab a first-in-class muscle-targeted therapy with the potential to establish a new standard of care in the treatment of spinal muscular atrophy, which is characterized by the irreversible loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle wasting that causes continuous motor function decline.

The European Medicines Agency is currently reviewing Scholar Rock's apitegromab application, with a decision expected near mid-2026.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

September 23, 2025 08:05 ET (12:05 GMT)