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), according to a news release published today.

The CRL is not specific to apitegromab’s safety, efficacy or third-party drug substance manufacturer. Instead, the CRL cited observations made during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility.

Scholar Rock, apitegromab’s developer, will resubmit the apitegromab BLA when Catalent Indiana addresses the FDA observations.

Apitegromab already has Orphan Drug Designation, Rare Pediatric Disease Designation, Priority Review and Fast Track Designation.

“While we are disappointed that the availability of a muscle-targeted treatment approac

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