Scholar Rock’s apitegromab has been cleared by the FDA for the treatment of spinal muscular atrophy—a key win for the company that culminates what has been a difficult regulatory path for the asset. The monoclonal antibody will be available to ship “in the coming days” under the brand name Isembyld.
Isembyld is the first muscle-targeted therapy that improves motor function in patients with spinal muscular atrophy (SMA), Scholar Rock said in a news release on Friday afternoon. The medicine is specifically indicated for patients two years and older who are also undergoing SMN2-targeted treatment. Friday’s approval came weeks earlier than the original target decision date of Sept. 30.