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Garetosmab

Phase 3

Fibrodysplasia Ossificans Progressiva | Small molecule | Other |Regeneron Pharmaceuticals, Inc.|Last Updated: Jan 16, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment63
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05394116A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)PHASE3 ACTIVE NOT_RECRUITING 63Nov 21, 2022Feb 27, 2029Jan 16, 202622 United States, Australia +16
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Study Endpoints
Primary Endpoints
Number of new HO lesions
At Week 56
Incidence and severity of treatment-emergent adverse events of special interest (AESIs)
Baseline to Week 56
Secondary Endpoints
Number of clinician-assessed flare-ups
Through Weeks 28, 56 and 84
Occurrence of new HO lesions
At Weeks 28, 56 and 84
Total volume of new HO lesions
At Weeks 28, 56 and 84
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
High dose GaretosmabEXPERIMENTALGaretosmab is administered by intravenous (IV) administration every 4 weeks (Q4W)
Low dose GaretosmabEXPERIMENTALGaretosmab is administered by IV administration Q4W
PlaceboEXPERIMENTALPlacebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W.
Interventions
NameTypeDescription
GaretosmabDRUGGaretosmab is supplied as a liquid drug product and will be administered IV.
PlaceboDRUGPlacebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites22

Key Inclusion Criteria: 1. Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) \[(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)\] 2. Confirmation of FOP diagnosis with documentation of Ty...

Countries:United StatesAustraliaBrazilChileChinaColombiaFinlandFranceHong KongItalyJapanMalaysiaNetherlandsPolandSouth AfricaSouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05394116primaryCompletionDate: changed
LOWMay 24, 2026NCT05394116studyFirstPostDate: changed