Dordaviprone
Dordaviprone is the first drug ever approved for a lethal childhood and young-adult brain tumour, diffuse midline glioma with the H3 K27M mutation (August 2025).
FDA accelerated approval 6 August 2025 for patients ≥1 year with H3 K27M-mutant diffuse midline glioma progressing after prior therapy, based on an integrated analysis of 50 patients across five trials: ORR 22%, median duration of response 10.3 months. Brain-penetrant oral agent from Oncoceutics → Chimerix → Jazz Pharmaceuticals (2025). Confirmatory phase 3 ACTION trial (newly diagnosed, after radiotherapy) ongoing. Debate continues on the strength of single-arm evidence.
- Notes
- No UK marketing authorisation as of September 2026, so no NICE appraisal. Access only through a clinical trial or a company early access scheme.
- NHS England
- Not routinely funded
Sources: NICE search: dordaviprone. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2025 | Recurrent H3 K27M-mutant diffuse midline glioma, age ≥1 (accelerated) |
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Latest papers
topQuery for this drug: (TITLE:"Dordaviprone" OR ABSTRACT:"Dordaviprone" OR TITLE:"Modeyso" OR ABSTRACT:"Modeyso" OR TITLE:"ONC201" OR ABSTRACT:"ONC201") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Dordaviprone, not a curated reading list.
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