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

Product

Active substance Tamibarotene
Domain Oncology and 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 comments Retinoic acid receptor (RAR) agonist.

Registration

Registration route Centralised (EMA)
Orphan drug Yes
Registration phase Clinical trials
Additional comments Fase II klinisch onderzoek

Therapeutic value

Therapeutic value No judgement
References NCT02807558
Additional comments Verschillende doseringen onderzocht

Expected patient volume per year

References NKR; LLS.org
Additional comments Totaal aantal AML patienten 674, 10-15% van de volwassen AML patienten heeft APL. Totaal aantal MDS patiënten betreft 695.

Expected cost per patient per year

References GoodRx.com
Additional comments 28 dose packs (3 tablets) Xermelo 250 mg $5,300.00 in de VS.

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.