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Dordaviprone is the first drug ever approved for a childhood and young-adult brain tumour that had none, 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.
Hyperactivates the mitochondrial protease ClpP and antagonises dopamine receptor D2, triggering integrated stress response and apoptosis in H3 K27M-altered cells.
1.Dordaviprone slips into a pocket on its target.
Background: ADC sequencing, Antigen escape (antigen loss, lineage switch), BCG-unresponsive, Castration-resistant prostate cancer (CRPC), Circulating tumour DNA (ctDNA). Also on OnCo: Resistance: how tumours escape each drug class · Lines of therapy.
Sources: NICE search: dordaviprone. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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Treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.
Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.1 years later, when the FDA's table was read. source
| Region | Year | Indication |
|---|---|---|
| US | 2025 | Recurrent H3 K27M-mutant diffuse midline glioma, age ≥1 (accelerated) |
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Query 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.
Shares H3 K27M (diffuse midline glioma), Blood-brain barrier (BBB), Diffuse midline glioma, H3 K27-altered (including DIPG), The brain: barrier and sanctuary.
Shares Develop drugs in children first when the target is a children's target, The brain: barrier and sanctuary, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Brain and spinal cord tumours (all types).
Shares Blood-brain barrier (BBB), The brain: barrier and sanctuary, Glioma & glioblastoma.
Shares Diffuse midline glioma, H3 K27-altered (including DIPG), Brain and spinal cord tumours (all types), Glioma & glioblastoma.
Shares Diffuse midline glioma, H3 K27-altered (including DIPG), Brain and spinal cord tumours (all types), Glioma & glioblastoma.
Shares H3 K27M (diffuse midline glioma), The brain: barrier and sanctuary, Glioma & glioblastoma.
Shares National Brain Tumor Society (NBTS), The brain: barrier and sanctuary, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Brain and spinal cord tumours (all types).
Shares H3 K27M mutation, Histone H3.3 (H3-3A), H3 K27M (diffuse midline glioma), Diffuse midline glioma, H3 K27-altered (including DIPG).