Enasidenib Manufacturers Plan to Submit Mutant IDH1 Inhibitor to FDA

Agios Pharmaceuticals announced plans to seek approval from the U.S. Food and Drug Administration (FDA) for its second leukemia drug – ivosidenib, a mutant IDH1 inhibitor – by the end of the year. Its first leukemia drug, enasidenib, a mutant IDH2 inhibitor, received FDA approval a few months ago.

The application will be supported by data from a clinical trial of 125 patients with relapsed or refractory IDH1-mutated acute myeloid leukemia (AML). Ivosidenib treatment led to complete remission (CR) rate of 21.6 percent. Another 8.8 percent of patients showed a complete response with partial hematologic recovery (CRh), for a combined CR/CRh rate of 30.4 percent. Median duration of response for these patients was 8.2 months.

Additional data from the ivosidenib clinical trial, including the percentage of AML patients who were able to become free of blood and platelet transfusions during treatment, will be presented at the 2017 American Society of Hematology Annual Meeting.

Source: STAT News, November 1, 2017.