New Medicines

Idhifa · Relapsed/refractory late stage acute myeloid leukaemia (AML) in patients with an IDH2 mutation


New molecular entity

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Approved (Licensed)

Jul 18: filed for approval in EU [9].

Aug 17: The drug will be priced at $24,872 per month (before discounts) in the US [8]

Aug 17. Approved in US [7]

Mar 17: Filed in the US for relapsed or refractory AML with certain mutations. Agios said it has been accepted under a priority review, with a decision set to be made by August 30. The NDA is based on a single arm phase 1/2 study of AG-221 in patients with advanced forms of blood cancer with an IDH2 mutation [6].

Oct 16: Celgene & Agios expect to submit a New Drug Application (NDA) to the FDA by the end of 2016 [5]; with Fast Track designation, this could potentially result in US licensing by the end of 2017.

Jul 16: In PIII development [1].

Apr 16: EMA grants orphan drug designation to AG 221 for treatment of AML [2].

Aug 14: US FDA grants fast track designation to AG 221 for treatment of patients with acute myeloid leukaemia that harbour an isocitrate dehydrogenase-2 (IDH2) mutation. AG 221 was also granted orphan drug designation by the US FDA in June 2014 [2].

Jun 14: AG 221 is being developed by Celgene under an exclusive worldwide license from Agios Pharmaceuticals [2].


First-in-class, small molecule, oral inhibitor of isocitrate dehydrogenase 2 (IDH2)
AML affects < 1.2 in 10,000 people in the EU (equivalent to not more than 61,000 people); overall UK age-standardised incidence rate in 2013 was 5 per 100,000 [4].
Relapsed/refractory late stage acute myeloid leukaemia (AML) in patients with an IDH2 mutation

Further information

To be confirmed

Trial or other data

Jul 16: IDHENTIFY study is currently recruiting patients. Collection of primary outcome data (overall survival) should complete Apr 19 [3].

Nov 15: Celgene initiates a PIII registration-enabling study to assess safety and efficacy of AG 221 in elderly patients with relapsed/refractory late-stage acute myeloid leukaemia (AML) with an IDH2 mutation (IDHENTIFY; AG221AML004; NCT02577406). The randomised, open-label trial is intended to enrol approximately 280 patients. Enrolment has begun in the US, and is expected to extend to Australia, Austria, Belgium, Canada, Denmark, France, Germany, Italy, Russia, South Korea, Spain and the UK [2].