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AUG 19 2026
UPCOMING · 77 DAYS TO PDUFA

Regeneron Pharmaceuticals garetosmab $REGN

for fibrodysplasia ossificans progressiva (FOP)

BLA · Priority Review · Orphan · Breakthrough

Accepted FEB 19 2026.

About

Fibrodysplasia ossificans progressiva (FOP) is an extremely rare genetic disorder where muscles and connective tissues gradually turn into bone, severely restricting movement over time. Garetosmab is an antibody designed to block activin A, a protein that drives this abnormal bone formation. By targeting this key biological signal, the drug may potentially slow the progression of debilitating skeletal immobilization in FOP patients.

Pivotal trial

OPTIMA Phase 3 active not recruiting NCT05394116

This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP)

Source: ClinicalTrials.gov

Also in development