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Tamibarotene

Amnolake

Extended indication Treatment of patients with relapsed or refractory non-APL AML (acute promyelocytenleukemie is een su
Therapeutic value No estimate possible yet
Registration phase Clinical trials

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)
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Fase 2 klinisch onderzoek

Therapeutic value

Therapeutic value No estimate possible yet
References NCT02807558
Additional remarks Verschillende doseringen onderzocht

Expected patient volume per year

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 dose packs (3 tablets) Xermelo 250mg $5,300 in de Verenigde Staten.

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

Indication extension No

Other information

There is currently no futher information available.