, Volume 77, Issue 15, pp 1705–1711 | Cite as

Enasidenib: First Global Approval

AdisInsight Report


Enasidenib (Idhifa®) is an oral isocitrate dehydrogenase-2 (IDH2) inhibitor developed by Celgene Corporation under a global, exclusive license from Agios Pharmaceuticals. Enasidenib has been approved in the USA for the treatment of adults with relapsed or refractory acute myeloid leukaemia (AML) and an IDH2 mutation as detected by an FDA-approved test. It is at various stages of development in other countries for AML, myelodysplastic syndromes and solid tumours. This article summarizes the milestones in the development of enasidenib leading to this first global approval in the USA for the treatment of adults with relapsed or refractory IDH2-mutated AML.


Compliance with Ethical Standards


The preparation of this review was not supported by any external funding.

Conflict of interest

During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the author on the basis of scientific completeness and accuracy. Esther Kim is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.

Additional information about this Adis Drug Review can be found at


  1. 1.
    Medeiros BC, Fathi AT, DiNardo CD, et al. Isocitrate dehydrogenase mutations in myeloid malignancies. Leukemia. 2017;31(2):272–81.CrossRefPubMedGoogle Scholar
  2. 2.
    Celgene Corporation. FDA grants approval of IDHIFA®, the first oral targeted therapy for adult patients with relapsed/refractory acute myeloid leukemia and an IDH2 mutation [media release]. 1 Aug 2017.
  3. 3.
    Döhner H, Weisdorf DJ, Bloomfield CD. Acute myeloid leukemia. N Engl J Med. 2015;373(12):1136–52.CrossRefPubMedGoogle Scholar
  4. 4.
    Celgene Corporation. IDHIFA® (enasidenib) tablets: US prescribing information. 2017. Accessed 30 Aug 2017.
  5. 5.
    US FDA. FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [media release]. 1 Aug 2017.
  6. 6.
    US FDA. List of cleared or approved companion diagnostic devices (in vitro and imaging tools). 2017. Accessed 22 Aug 2017.
  7. 7.
    Adis Insight. Drug profile: enasidenib companion diagnostic. 2017. Accessed 22 Aug 2017.
  8. 8.
    Adis Insight. Drug profile: enasidenib. 2017. Accessed 22 Aug 2017.
  9. 9.
    Cardinale L, Asteggiano F, Moretti F, et al. Pathophysiology, clinical features and radiological findings of differentiation syndrome/all-trans-retinoic acid syndrome. World J Radiol. 2014;6(8):583–8.CrossRefPubMedPubMedCentralGoogle Scholar
  10. 10.
    Agios Pharmaceuticals. Celgene Corporation and Agios Pharmaceuticals announce global strategic collaboration to advance unique science of cancer metabolism [media release]. 15 Apr 2010.
  11. 11.
    Agios Pharmaceuticals. Celgene and Agios extend cancer metabolism collaboration [media release]. 5 Oct 2011.
  12. 12.
    Agios Pharmaceuticals. Agios advances cancer metabolism collaboration with Celgene [media release]. 11 Dec 2013.
  13. 13.
    Agios Pharmaceuticals. Agios Pharmaceuticals announces that Celgene exercised its option to license AG-221 under global strategic collaboration [media release]. 13 Jun 2014.
  14. 14.
    Agios Pharmaceuticals. Agios announces Celgene decision to extend discovery phase of global strategic collaboration to April 2016 [media release]. 8 Dec 2014.
  15. 15.
    Agios Pharmaceuticals. Agios and Celgene establish new collaboration in metabolic immuno-oncology and amend certain rights from 2010 agreement [media release]. 17 May 2016.
  16. 16.
    Amatangelo MD, Quek L, Shih A, et al. Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response. Blood. 2017;130(6):732–41.CrossRefPubMedPubMedCentralGoogle Scholar
  17. 17.
    Stein EM, DiNardo CD, Pollyea DA, et al. Enasidenib in mutant-IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017;130(6):722–31.CrossRefPubMedPubMedCentralGoogle Scholar
  18. 18.
    National Library of Medicine. 2017. Accessed 17 Aug 2017.
  19. 19.
    Agios Pharmaceuticals. New data from phase 1/2 trial of oral IDHIFA® (enasidenib) demonstrate durable complete responses in patients with IDH2 mutant relapsed or refractory AML [media release]. 24 Jun 2017.
  20. 20.
    Agios Pharmaceuticals. Agios announces data from ongoing phase 1/2 trial of AG-221 showing durable responses in patients with advanced hematologic malignancies [media release]. 6 Dec 2015.
  21. 21.
    Celgene Corporation. Celgene and Agios announce collaborations with Abbott for diagnostic identification of IDH mutations in AML [media release]. 12 Oct 2016.
  22. 22.
    Agios Pharmaceuticals. Agios Pharmaceuticals announces initiation of a phase 1/2 clinical trial of AG-221 in patients with advanced solid tumors with an IDH2 mutation [media release]. 21 Oct 2014.
  23. 23.
    Tallman MS, Knight RD, Glasmacher AG, et al. Phase III randomized, open-label study comparing the efficacy and safety of AG-221 vs conventional care regimens (CCR) in older patients with advanced acute myeloid leukemia (AML) with isocitrate dehydrogenase (IDH)-2 mutations in relapse or refractory to multiple prior treatments: the IDHENTIFY trial [abstract no. TPS7074]. J Clin Oncol. 2016;34(15 Suppl).Google Scholar
  24. 24.
    Agios Pharmaceuticals. Agios announces initiation of phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA® (azacitadine for injection) in newly diagnosed acute myeloid leukemia (AML) patients not eligible for intensive chemotherapy [media release]. 30 Mar 2016.
  25. 25.
    Agios Pharmaceuticals. Agios announces initiation of phase 1b frontline trial of AG-221 or AG-120 in combination with intensive chemotherapy in newly diagnosed acute myeloid leukemia (AML) patients [media release]. 18 Dec 2015.

Copyright information

© Springer International Publishing AG 2017

Authors and Affiliations

  1. 1.SpringerAucklandNew Zealand

Personalised recommendations