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Diffuse midline glioma | Treatment | Targeted therapy | Dordaviprone (ONC201 etc.)
Home > Publications > Topics > Diffuse midline glioma > Treatment > Targeted therapy > Dordaviprone (ONC201)
Hansford JR, Manoharan N, Bouche G, Ramaswamy V, Jabado N, Bouffet
E.
ONC201 (Dordaviprone): review of
evidence to date in diffuse midline glioma, hope or hype?
Expert
Opin Emerg Drugs. 2024 Nov 21:1-5. doi:
10.1080/14728214.2024.2426649. PMID:
39548664. Perspective. ˍ
Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE,
Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot
J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A,
Kilburn L, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS,
Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore R, Taylor
L, Umemura Y, Wen PY.
ONC201
(Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline
Glioma.
J
Clin Oncol. 2024 Feb 9:JCO2301134. doi:
10.1200/JCO.23.01134. PMID:
38335473. Interventional study. ˍ
Di Carlo D, Annereau M, Vignes M, Denis L, Epaillard N, Dumont S,
Guyon D, Rieutord A, Jacobs S, Salomon V, Yoldjian I, Duperray F,
Brunel L, Baiao X, Lemos F, Vauleon E, Capra M, Abbou S, Touat M,
Sanson M, Gandemer V, De Carli E, Bourdeaut F, Hezam I, Vassal G,
Grill J.
Real life data of ONC201
(dordaviprone) in pediatric and adult H3K27-altered recurrent
diffuse midline glioma: Results of an international
academia-driven compassionate use program.
Eur
J Cancer. 2024 Dec 11;216:115165. doi:
10.1016/j.ejca.2024.115165. PMID:
39700833. Compassionate use. ˍ
Faison SL, Batonga J, Arumugham T, Bartkus A, Morrison M, Mullin
MJ, Tippin T, Naderer O.
A Phase 1
Randomized Study to Evaluate the Safety, Tolerability, and
Pharmacokinetics of Single Escalating Oral Doses of Dordaviprone
and the Effects of Food on the Bioavailability of Dordaviprone in
Healthy Adult Subjects.
Clin
Pharmacol Drug Dev. 2025 Jan 16. doi:
10.1002/cpdd.1512. PMID:
39817482. Interventional study. ˍ
Faison SL, Batonga J, Arumugham T, Bartkus A, Morrison ME, Mullin
MJ, Tippin T, Naderer O.
Effect of
Severe Renal Impairment on Dordaviprone (ONC201) Pharmacokinetics.
Drugs
R D. 2025 Jul 30. doi:
10.1007/s40268-025-00520-x. PMID:
40742663. Interventional study.
ˍ
Timpanaro A, Song EZ, Toumi R, Elena-Sanchez L, Meechan M, Piccand
C, Nemec K, Kordowski A, Lau D, Johnson S, Winter L, Rajendran A,
Ronsley R, Oda SK, Gustafson J, Wendler JP, Koschmann C, Evans M,
Pattwell S, Jensen MC, Foster JB, Dun MD, Biery MC, Vitanza NA.
Preclinical efficacy of combinatorial
B7-H3 CAR T cells and ONC206
against diffuse intrinsic pontine glioma.
bioRxiv
[Preprint]. 2025 Sep 4:2025.09.01.673023. doi:
10.1101/2025.09.01.673023. PMID:
40950080. Lab investigation. ˍ
Zafar S, Furqan M, Binte Rahim S, Haider MU.
Dordaviprone
in H3K27M-mutant diffuse midline glioma: an editorial on emerging
targeted therapy.
Ann
Med Surg (Lond). 2025 Oct 13;87(12):7886-7888. doi:
10.1097/MS9.0000000000004079. PMID:
41377309. Editorial.
ˍ
Lawrence L.
Diffuse midline gliomas
get first FDA-approved drug.
Cancer. 2025 Dec
17;131(24):e70146. doi: 10.1002/cncr.70146. PMID: 41405355. News.
ˍ
Woo PYM, Lee CKW, Li LF, Hui VKH, Chan DTM, Lee MWY, Chan TKT,
Wong DKK, Chow JSW, Ho JMK, Chow JKH, Tse TPK, Ko NMW, Cheung KM,
Leung AKC, Wong KCW, Loong HHF, Poon WS, El-Helali A.
Outcomes
of H3K27M-altered diffuse midline glioma adult Chinese patients
and real-world experience with German-sourced ONC201 targeted
therapy: A multi-center study in Hong Kong.
Neurooncol
Adv. 2026 Mar 21;8(1):vdag074. doi:
10.1093/noajnl/vdag074. PMID:
42011291. ˍ
Khasraw M, Dun MD, Arrillaga-Romany I, Verhaak RGW, Bouffet E,
Hansford JR, Singh K, Mueller S, Koschmann C, Wen PY.
Dordaviprone
for Diffuse Midline Glioma: A Landmark Approval or a Premature
Step?
Neuro
Oncol. 2026 May 22:noag117. doi:
10.1093/neuonc/noag117. PMID:
42172613. ˍ