Ivosidenib

Orphan DrugFDA Approved

Description

Ivosidenib is an isocitrate dehydrogenase-1 (IDH1) inhibitor used in the treatment of acute myeloid leukemia (AML) with a susceptible IDH1 mutation. While not directly indicated for classic neuroendocrine tumor of the appendix, IDH1 mutations can occur in various cancers, and ivosidenib may be explored in clinical trials if such a mutation is present.

Indications & Therapeutic Use

Acute Myeloid Leukemia (AML) with IDH1 mutation

Global Availability (1 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Ivosidenib
Generic NameIvosidenib
Brands1 brand available
Active IngredientIvosidenib
Drug ClassAcute Myeloid Leukemia (AML) with IDH1 mutation
ManufacturerServier Pharmaceuticals
Dosage FormsTablets
Medical CodeL01XE30
Orphan StatusYes — Orphan Drug
Cold ChainNot Required
Lead Time14 days
Reg. StatusFDA Approved
Clinical TrialNCT02074839
Countries1 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes