Scholar Rock Drug Wins First-in-Class Approval in Rare Neuromuscular Disease
News from MedCity News: Scholar Rock Drug Wins First-in-Class Approval in Rare Neuromuscular Disease
Scholar Rock
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Its lead product, apitegromab, is being developed for spinal muscular atrophy (SMA).
Company Info
Sector:Biopharmaceuticals
Headquarters:Cambridge, Massachusetts, United States
Founded:2012
Website:scholarrock.com
Upcoming Milestones & Key Dates
Late 2026
Regulatory
Apitegromab BLA submission decision
Q4 2026
Clinical
SRK-439 Phase 1 initial data
Clinical Development Pipeline
Apitegromab
Target: Latent myostatinSRK-439
Target: GDF11/GDF8SRK-181
Target: Latent TGFβ1Recent News
Scholar Rock secures first FDA approval for muscle-targeting SMA treatment
Scholar Rock has received FDA approval for its muscle-targeting treatment for spinal muscular atrophy, marking the company first regulatory approval.
Scholar Rock drops Novo manufacturing site from SMA drug application
Scholar Rock drops Novo manufacturing site from SMA drug application
Scholar Rock rejigs approval filing after FDA flags violations at Novo plant
Despite earlier claims of improvement at the fill-finish plant, the FDA has classified the facility as Official Action Indicated based on inspection findings.
Scribe's $96M IPO; Scholar Rock awaits site classification for apitegromab
Scribe's $96M IPO; Scholar Rock awaits site classification for apitegromab.
Leadership Team
David L. Hallal
Chief Executive Officer
Mo Qatanani
Chief Scientific Officer
Tracy Wong
Chief Financial Officer