FDA Accepts sNDA for AML Therapy

The FDA’s acceptance of the application paves the way for an early review time.

The FDA has accepted a supplemental New Drug Application (sNDA) for ivosidenib (Tibsovo, Agios Pharmaceuticals) for patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy.1

The sNDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of June 21, 2019. The FDA’s Priority Review status expedites the review time from the initial 10 months to a potential 6 months from the day of filing acceptance.1 It is typically given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists.

According to the press release, the FDA accepted the ivosidenib sNDA under its Real-Time Oncology Review pilot program, whose goal is to streamline reviews for oncology drugs by allowing the agency access to clinical trial data before a formal submission.

AML begins in the myeloid, or bone marrow, cells, but frequently transmits into the blood. The fast-growing cells can potentially spread to other parts of the body including lymph nodes, liver, spleen, central nervous system, and testicles, according to the American Cancer Society.2

Ivosidenib is a first-in-class, oral, targeted inhibitor of mutant IDH1. According to the press release, the sNDA submission is based on results from the untreated AML patients from the Phase 1 dose-escalation and expansion study of ivosidenib in patients with newly diagnosed AML ineligible for standard treatment.1


  • What is Acute Myeloid Leukemia (AML)? American Cancer Society. https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html. Accessed Feb. 21, 2019.
  • Agios Announces FDA Acceptance of Supplemental New Drug Application for TIBSOVO® (ivosidenib) for the Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation Not Eligible for Standard Therapy [news release]. Cambridge, Massachusetts. February 20, 2019: GlobeNewswire. https://globenewswire.com/news-release/2019/02/20/1738209/0/en/Agios-Announces-FDA-Acceptance-of-Supplemental-New-Drug-Application-for-TIBSOVO-ivosidenib-for-the-Treatment-of-Patients-with-Newly-Diagnosed-Acute-Myeloid-Leukemia-with-an-IDH1-Mu.html. Accessed Feb. 21, 2019.