Olutasidenib

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
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
ATMP	No
Submission date	2024
Expected Registration	2025
Registration phase	Clinical trials

Therapeutic value

Therapeutic value	No estimate possible yet
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.

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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.