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Dordaviprone

Phase 3

H3 K27M | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Apr 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-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment510
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05580562ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)PHASE3 RECRUITING 510Jan 23, 2023Jun 1, 2028Apr 16, 2026162 United States, Argentina +16
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Study Endpoints
Primary Endpoints
Overall survival (OS)
From date of randomization until date of death from any cause, assessed up to approximately 44 months

Overall Survival is defined as the time from randomization to death due to any cause.

Secondary Endpoints
Progression Free Survival (PFS) using RANO 2.0 Criteria for All Participants
From date of randomization until the date of first documented progression assessed up to approximately 44 months.
PFS Using RANO 2.0 Criteria for Participants with Measurable Contrast-Enhancing Disease
From date of randomization up to 44 months
Incidence of adverse events
From date of randomization up to 44 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Dordaviprone Twice Weekly GroupEXPERIMENTAL -
Dordaviprone Once Weekly GroupEXPERIMENTAL -
Placebo GroupPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
Dordaviprone (ONC201)DRUGParticipants ≥ 52.5 kg will receive 625 mg of dordaviprone (5 × 125-mg capsules) dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments.
Dordaviprone (ONC201) + PlaceboDRUGParticipants ≥ 52.5 kg will receive 625 mg of dordaviprone (5 × 125-mg capsules) or matching placebo on dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments
PlaceboOTHERParticipants will receive placebo (same number of capsules as the dordaviprone dose) on dosing days
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites162

Inclusion Criteria: 1. Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable. 2. Body weight ≥ 10 kg at time of randomization. 3. Histologically diagnosed ...

Countries:United StatesArgentinaAustraliaAustriaBrazilCanadaDenmarkGermanyHong KongIsraelItalyJapanNetherlandsSingaporeSouth KoreaSpainSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05580562Enrollment: 450 → 510
LOWMay 24, 2026NCT05580562studyFirstPostDate: changed