Version 4

Olutasidenib

Medicine

Extended indication Patients with AML or MDS with an IDH1 mutation.
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Olutasidenib
Domain Hematology
Reason of inclusion New medicine (specialité)
Main indication AML / MDS
Extended indication Patients with AML or MDS with an IDH1 mutation.
Manufacturer Rigel
Mechanism of action Unknown
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Isocitrate dehydrogenase 1 inhibitors.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity Unknown
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug No
Registration phase Clinical trials
Additional remarks Het is nog niet bekend wanneer de firma een MAA gaat indienen bij de EMA.

Therapeutic value

Therapeutic value No estimate possible yet

This assessment does not indicate any potential inclusion in the package.
Substantiation De therapeutische waarde is nog onduidelijk.
Additional remarks NCT02719574

Expected patient volume per year

Patient volume 86 - 119

Market share is generally not included unless otherwise stated.
References NKR2021 (1) J Natl Cancer Inst. 2010 Jul 7; 102(13): 932–941. doi: 10.1093/jnci/djq187. Walter et al., Leukemia. 2015 Feb; 29(2): 312–320 (2) Thol F, Weissinger EM, Krauter J, Wagner K, Damm F, Wichmann M, Göhring G, Schumann C, Bug G, Ottmann O, Hofmann WK, Schlegelberger B, Ganser A, Heuser M. IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica. 2010 Oct;95(10):1668-74. doi: 10.3324/haematol.2010.025494. Epub 2010 May 21. PMID: 20494930; PMCID: PMC2948091 (3)
Additional remarks Er waren 853 AML diagnoses in 2021 (1). Van de AML patiënten heeft 6% tot 10% een IDH1mutatie (2) (52 tot 84 patiënten). In 2021 waren er in totaal 953 MDS diagnoses. Van de MDS patiënten heeft ongeveer 3,6% een IDH1 mutatie (3). Dit betekent een verwacht patiëntvolume van 34. In totaal betekent dit een patiëntvolume van 86 tot 119.

Expected cost per patient per year

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions Unknown

Other information