Community update from Scholar Rock: FDA approval of ISEMBYLD™ (apitegromab-mstn)
Scholar Rock announced on 11 September 2026 that the U.S. Food and Drug Administration (FDA) has approved apitegromab (Isembyld) for the treatment of SMA in adults and children aged 2 years and older who are receiving an SMN2-targeted treatment.
Apitegromab is the first muscle-targeted treatment to become available to the SMA community.
As previously shared, following the U.S. FDA’s classification of an inspection of one of the company’s third-party fill-finish facilities as Official Action Indicated, in alignment with the European Medicines Agency (EMA), Scholar Rock has withdrawn its European Marketing Authorisation Application. The company plans to resubmit the application using an alternative facility, with the aim of progressing the European review as quickly as possible.
SMA Europe is in close contact with Scholar Rock and will share further information on the European marketing authorisation as soon as it becomes available.