Garetosmab garetosmab PHASE3
Drug Profile
ModalityMonoclonal antibody
RouteIV
Therapy AreaRare Disease
Peak Sales Est$500M
Formulations[]
Companies
REGN (ORIGINATOR)100%
Mechanism: Activin A inhibitor (monoclonal antibody)
Expert: Fully human IgG1 monoclonal antibody targeting activin A. In FOP, activin A signals through the mutant ACVR1 (ALK2 R206H) receptor, triggering heterotopic ossification. Blocking activin A prevents pathological bone formation.
Everyday: Blocks activin A, the signal that causes muscles and connective tissue to turn into bone in FOP patients. Activin A is the "wrong signal" that triggers bone growth where it should not happen — this drug stops that signal.
Targets: ["Activin A"]
Programs (1)
IndicationStageKey StudyRegional Status
FOPPHASE3OPTIMA[]
Notes
Anti-activin A antibody for FOP. Only ~900 patients worldwide with confirmed FOP. LUMINA-1 Ph2 showed 54% reduction in new heterotopic bone volume vs placebo. Breakthrough Therapy Designation. No approved treatments exist for FOP. Ph3 (OPTIMA) ongoing.
Data from Supabase · Updated 2026-03-24