Garetosmab

Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal dominant disorder of episodic, progressive heterotropic ossification. FOP is characterized by episodic inflammatory episodes, which may be precipitated by trauma, including immunizations and minor tissue damage, which usually result in ossification of the lesion. Patients experience abnormal cartilage formation, growth plate dysplasia, and joint issues, resulting in progressive immobility and associated comorbidities. The discovery of an activating mutation in the ACVR1 receptor that renders it responsive to the (normally antagonistic) Activin A led to an interest in Activin A as a therapeutic target.

Garetosmab is under investigation in clinical trial NCT04577820 (Study to Assess the Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans Progressiva (FOP)).