Scholar Rock Gets FDA Fast Track, Orphan Drug Designations for Muscular Disorder Drug

08:23 AM EDT, 09/02/2026 (MT Newswires) -- Scholar Rock (SRRK) said Wednesday that the US Food and Drug Administration granted fast-track and orphan drug designations to its experimental drug apitegromab for facioscapulohumeral muscular dystrophy. The company said it has started dosing adults in a phase 2 trial evaluating apitegromab in patients with genetically confirmed facioscapulohumeral muscular dystrophy. The study is expected to enroll about 60 participants, who will receive either apitegromab or a placebo every four weeks for 52 weeks, the company said, adding that the main goal is to measure changes in total lean muscle volume using MRI at Week 52. Scholar Rock said the fast track designation is intended to speed the development and review of drugs for serious conditions with significant unmet medical needs.