Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare inherited disorder in which muscle and connective tissues progressively ossify, forming extra-skeletal bone and causing severe movement restriction. The condition usually begins in early childhood and can be worsened by trauma or inflammatory flare-ups. Pathogenic variants in ACVR1 are a key cause.
The FDA has accepted Regeneron's Biologics License Application for garetosmab to treat fibrodysplasia ossificans progressiva, granting Priority Review with a target action date of August 2026. The application is supported by Phase 3 OPTIMA trial data.
The FDA has accepted Regeneron's biologics license application for garetosmab with priority review, targeting fibrodysplasia ossificans progressiva. A decision is expected by August 2026 based on Phase 3 trial data showing significant reductions in new bone lesions.
Regeneron said the FDA accepted its garetosmab BLA for FOP and granted Priority Review, with a decision expected by August 2026. Phase 3 OPTIMA data reported major lesion reductions versus placebo.
The FDA has accepted for Priority Review Regeneron's biologics license application for garetosmab to treat adults with fibrodysplasia ossificans progressiva, with a target action date of August 2026.
The FDA has accepted for Priority Review the Biologics License Application for garetosmab to treat adults with fibrodysplasia ossificans progressiva, with a target decision date of August 2026. The application is supported by Phase 3 OPTIMA trial data showing significant reductions in heterotopic bone lesions.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06508021 |
A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP) |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT06089616 |
A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP) |
RECRUITING | |
| NCT05394116 |
A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05039515 |
A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). |
TERMINATED | PHASE2 |
| NCT04829773 |
Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects |
COMPLETED | PHASE1 |
| NCT04818398 |
Study of Single-Ascending Doses of DS-6016a in Healthy Japanese Subjects |
COMPLETED | PHASE1 |
| NCT04665323 |
An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families. |
COMPLETED | |
| NCT04307953 |
Saracatinib Trial TO Prevent FOP |
RECRUITING | PHASE2 |
| NCT03312634 |
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva. |
COMPLETED | PHASE3 |
| NCT03188666 |
A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva |
COMPLETED | PHASE2 |
