Oral treatment for patients with relapsed or refractory acute myeloid leukemia filed at FDA.

Agios Pharmaceuticals, Inc. a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, announced that it has submitted a New Drug Application (NDA) to the FDA for ivosidenib (AG 120), an investigational oral treatment for patients with relapsed or refractory acute myeloid leukemia (R/R AML) and an isocitrate dehydrogenase-1 (IDH1) mutation. Agios has requested priority review for the application, which, if granted, could result in a six-month review process.

The NDA is supported by data from the ongoing Phase 1 dose-escalation and expansion study of ivosidenib in patients with advanced hematologic malignancies and an IDH1 mutation. Ivosidenib is wholly owned by Agios. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.

Comment: Data from the Phase I trial was presented at an oral session at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition which demonstrated a complete response (CR) and CR with partial hematologic recovery (CRh) rate of 30.4% and an overall response rate (ORR) of 41.6% in the primary analysis set of 125 patients with R/R AML who received ivosidenib at 500 mg once daily and received their first dose at least 6 months prior to the May 12, 2017 analysis cutoff date.