Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Garetosmab · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Adolescents | EXPERIMENTAL | - |
| Cohort 2: Children | EXPERIMENTAL | - |
| Cohort 3: Children and Adolescents | EXPERIMENTAL | - |
| High dose Garetosmab | EXPERIMENTAL | Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W) |
| Low dose Garetosmab | EXPERIMENTAL | Garetosmab is administered by IV administration Q4W |
| Placebo | EXPERIMENTAL | Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W. |
| Name | Type | Description |
|---|---|---|
| garetosmab | DRUG | Administered per the protocol |
| Placebo | DRUG | Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV. |
Key Inclusion Criteria: 1. For USA participants, age criteria are 4 to \< 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to \< 18 years old 2. Must have a confirmation of FOP diagnosis, as described in the protocol 3....
Garetosmab is an investigational drug being developed for the treatment of Fibrodysplasia Ossificans Progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic bone formation. It is currently in Phase 3 clinical trials for this condition.
Garetosmab is a monoclonal antibody (mab) that targets a specific molecular pathway involved in FOP. As an antibody, it is designed to bind to and modulate its target to potentially slow or prevent abnormal bone growth in patients with FOP.
Garetosmab is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company. Regeneron is conducting clinical trials to evaluate the safety and efficacy of Garetosmab in patients with Fibrodysplasia Ossificans Progressiva.
Garetosmab is currently in Phase 3 clinical development. It is being studied in adult patients with Fibrodysplasia Ossificans Progressiva in a randomized, double-blind, placebo-controlled trial. It is also being investigated in a pediatric trial for the same condition.
Garetosmab is being evaluated in two Phase 3 trials. The first, NCT05394116, is an active, not recruiting study in adults with FOP, enrolling 63 participants across multiple countries. The second, NCT07559513, is a not yet recruiting study in children and adolescents with FOP, with a planned enrollment of 18 participants.
Garetosmab has received Priority Review designation from the FDA, but it is not yet approved. It is still in clinical development, with ongoing Phase 3 trials assessing its safety and efficacy in patients with Fibrodysplasia Ossificans Progressiva.