Scholar Rock (NASDAQ:SRRK) shares fell 14% on Tuesday following the U.S. Food and Drug Administration’s (FDA) issuance of a Complete Response Letter (CRL) regarding the company’s spinal muscular atrophy (SMA) treatment, apitegromab.
The FDA’s concerns stemmed exclusively from findings during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility recently acquired by Novo Nordisk. Scholar Rock emphasized that the observations were not linked specifically to apitegromab and that the CRL did not raise any additional issues regarding the drug’s safety or efficacy.
Scholar Rock intends to resubmit its Biologics License Application (BLA) for apitegromab once Catalent Indiana resolves the FDA’s manufacturing observations. The company noted that Catalent submitted a detailed response in early August and has been actively implementing corrective measures.
“We are continuing to work closely with Catalent Indiana on the FDA’s manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible,” said David L. Hallal, Chairman and CEO of Scholar Rock.
Apitegromab has received several FDA special designations, including Orphan Drug Designation, Rare Pediatric Disease Designation, Priority Review, and Fast Track, underscoring its potential to address unmet needs in SMA therapy.
Outside the United States, the European Medicines Agency (EMA) is reviewing apitegromab’s marketing authorization application, with a decision anticipated around mid-2026. If approved, the drug could launch in Europe in the second half of 2026, with Germany likely to offer patient access first.
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.