Dordaviprone
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| Trade names | Modeyso |
| Other names | ONC201, ONC-201 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625097 |
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| Routes of administration | By mouth |
| Drug class | Protease activator |
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| Formula | C24H26N4O |
| Molar mass | 386.499 g·mol−1 |
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Dordaviprone, sold under the brand name Modeyso, is an anti-cancer medication used for the treatment of diffuse midline glioma (a type of brain tumor).[1][2] Dordaviprone is a protease activator of the mitochondrial caseinolytic protease P.[1] It is dopamine receptor D2 antagonist and an allosteric activator of the mitochondrial caseinolytic protease P.[3]
Dordaviprone was approved for medical use in the United States in August 2025.[2] It is the first approval of a systemic therapy for H3 K27M-mutant diffuse midline glioma by the US Food and Drug Administration (FDA).[2] The FDA considers it to be a first-in-class medication.[4][5]
Medical uses
[edit]Dordaviprone is indicated for the treatment of people with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.[1][2]
History
[edit]Efficacy was evaluated in an integrated efficacy population of 50 participants with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label, non-randomized clinical trials conducted in the US (ONC006 [NCT02525692], ONC013 [NCT03295396], ONC014 [NCT03416530], ONC016 [NCT05392374], and ONC018 [NCT03134131]).[2] The efficacy population comprised participants who received single-agent dordaviprone for diffuse midline glioma harboring an H3 K27M mutation and had progressive and measurable disease per Response Assessment in Neuro-Oncology-High Grade Glioma (RANO-HGG) criteria.[2] Participants were also at least 90 days post radiation therapy, had an adequate washout period from prior anticancer therapies, a Karnofsky Performance Status/Lansky Performance Status (KPS/LPS) score ≥ 60, and stable or decreasing corticosteroid use.[2] Participants with diffuse intrinsic pontine glioma, primary spinal tumors, atypical histologies, or cerebrospinal fluid dissemination were excluded.[2]
The US Food and Drug Administration granted the application for dordaviprone priority review, orphan drug, rare pediatric disease, and fast track designations.[2]
Society and culture
[edit]Legal status
[edit]Dordaviprone was approved for medical use in the United States in August 2025.[2][6]
Names
[edit]Dordaviprone is the international nonproprietary name.[7]
Dordaviprone is sold under the brand name Modeyso.[2][6]
Dordaviprone was previously known as ONC201, and even earlier, TIC10, during its developmental phase. [8]
References
[edit]- 1 2 3 4 "Modeyso- dordaviprone capsule". DailyMed. 9 January 2026. Retrieved 22 July 2026.
- 1 2 3 4 5 6 7 8 9 10 11 "FDA grants accelerated approval to dordaviprone for diffuse midline glioma". U.S. Food and Drug Administration (FDA). 6 August 2025. Retrieved 7 August 2025.
This article incorporates text from this source, which is in the public domain. - ↑ Prabhu VV, Morrow S, Rahman Kawakibi A, Zhou L, Ralff M, Ray J, et al. (December 2020). "ONC201 and imipridones: Anti-cancer compounds with clinical efficacy". Neoplasia. 22 (12). New York, N.Y.: 725–744. doi:10.1016/j.neo.2020.09.005. PMC 7588802. PMID 33142238.
- ↑ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 8 July 2026. Retrieved 19 July 2026.
- ↑ "Advancing Health Through Innovation: New Drug Therapy Approvals 2025" (PDF). U.S. Food and Drug Administration (FDA). January 2026.
- 1 2 "Jazz Pharmaceuticals Announces U.S. FDA Approval of Modeyso (dordaviprone) as the First and Only Treatment for Recurrent H3 K27M-mutant Diffuse Midline Glioma" (Press release). Jazz Pharmaceuticals. 6 August 2025. Retrieved 10 August 2025 – via PR Newswire.
- ↑ World Health Organization (2023). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 89". WHO Drug Information. 37 (1). hdl:10665/366661.
- ↑ https://doi.org/10.1016/j.gendis.2026.1023357
External links
[edit]- Clinical trial number NCT02525692 for "Oral ONC201 in Adult Recurrent Glioblastoma" at ClinicalTrials.gov
- Clinical trial number NCT03295396 for "ONC201 in Adults With Recurrent H3 K27M-mutant Glioma" at ClinicalTrials.gov
- Clinical trial number NCT03416530 for "ONC201 in Pediatric H3 K27M Gliomas" at ClinicalTrials.gov
- Clinical trial number NCT05392374 for "Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas" at ClinicalTrials.gov
- Clinical trial number NCT03134131 for "Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas" at ClinicalTrials.gov
