FDA dishes out CRL for Scholar Rock’s apitegromab

US biotech Scholar Rock’s (Nasdaq: SRRK) saw its shares tumble almost 12% to $28.77 in pre-market activity, after it announced a regulatory setback from the US Food and Drug Administration (FDA).

The company revealed that the FDA has issued a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of patients with spinal muscular atrophy (SMA).

The CRL is related to observations identified during a routine general site inspection o Catalent Indiana, a third-party fill-finish facility which was acquired by Novo Nordisk (NOV: N) in December 2024. The observations are not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab’s efficacy and safety data or the third-party drug substance manufacturer.