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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