Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of people living with spinal muscular atrophy (SMA).
The CRL is related to observations identified during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility, and is not specific to apitegromab. Importantly, the FDA did not cite any other approvability concerns related to apitegromab’s efficacy, safety data, or the third-party drug substance manufacturer.
Scholar Rock has stated its intention to resubmit the apitegromab BLA once Catalent Indiana has successfully resolved the FDA’s observations. The company believes that the FDA will be able to act expeditiously on the application following resubmission.
While this is disappointing news, the issues identified by the regulator appear to be due to an unrelated manufacturing plant issue rather than a problem with the clinical data or apitegromab itself. Cure SMA will be working with the FDA and Scholar Rock to identify a timeline for the resolution of these issues and will communicate more information to the community soon.
To read Scholar Rock's full community statement, please click here.