Version 4

Tamibarotene

Medicine

Extended indication Treatment of patients with relapsed or refractory non-APL AML (acute promyelocytenleukemie is een subtype van acute myeloïde leukemie), relapsed or re
Therapeutic value No estimate possible yet
Registration phase No registration expected

Product

Active substance Tamibarotene
Domain Hematology
Reason of inclusion New medicine (specialité)
Main indication AML / MDS
Extended indication Treatment of patients with relapsed or refractory non-APL AML (acute promyelocytenleukemie is een subtype van acute myeloïde leukemie), relapsed or refractory higher-risk MDS (myelodysplastic syndrome), newly diagnosed treatment naïve AML, or transfusion dependent, lower-risk MDS.
Proprietary name Amnolake
Manufacturer Syros
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Retinoic acid receptor (RAR) agonist.

Registration

Registration route Centralised (EMA)
Type of trajectory Unknown
Particularity Unknown
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug Yes
Registration phase No registration expected
Additional remarks De studie is verwijderd uit de pijplijn van de fabrikant en er is geen recente ontwikkeling gemeld.

Therapeutic value

Therapeutic value No estimate possible yet

This assessment does not indicate any potential inclusion in the package.
References NCT02807558
Additional remarks Verschillende doseringen onderzocht.

Expected patient volume per year

Patient volume

Market share is generally not included unless otherwise stated.
References NKR; LLS.org
Additional remarks Totaal aantal AML patiënten 674, 10 tot 15% van de volwassen AML patiënten heeft APL. Totaal aantal MDS patiënten betreft 695.

Expected cost per patient per year

References GoodRx.com
Additional remarks 28 dosisverpakking (3 tabletten) Xermelo 250mg $5,300 in de Verenigde Staten.

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions No

Other information