Today, Scholar Rock announced that it has resubmitted its Biologics License Application (BLA) to the Food and Drug Administration (FDA) for apitegromab, an investigational therapy for the treatment of people living with spinal muscular atrophy (SMA). Apitegromab is an anti-myostatin muscle-targeted treatment.
The resubmission follows a previous Complete Response Letter (CRL) issued by the FDA related to observations identified during a routine inspection of a third-party fill-finish manufacturing facility, Catalent Indiana LLC. These observations were not specific to apitegromab, and importantly, the FDA did not raise concerns regarding the therapy’s efficacy, safety data, or the drug substance manufacturer.
Scholar Rock has since worked closely with the FDA and the manufacturing partner to address these observations. This included participation in a Type A meeting with the FDA, as well as follow-up discussions between the FDA and Catalent Indiana. Based on these interactions, Scholar Rock aligned with the FDA to proceed with resubmission of the BLA prior to reinspection of the facility.
Importantly, as part of the resubmission, Scholar Rock has also included a second U.S.-based fill-finish manufacturing facility to strengthen supply chain reliability and support future demand.
Scholar Rock anticipates FDA acceptance of the BLA within 30 days of resubmission and a review period of up to six months from the date of resubmission.
Cure SMA will continue to monitor progress and remain in close communication with Scholar Rock and the FDA. We will share updates with the community as more information becomes available. If you have questions, please reach out to us at [email protected].
You can also visit our updated FAQ page for answers to common questions related to this news.