The New Drug Application (NDA) for enasidenib (AG-221/CC-90007) has been accepted by the FDA for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation. The 5-year survival rate for AML is approximately 20-25 percent. IDH2 mutations are present in about 8-19 percent of AML cases.
The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act action date of Aug. 30, 2017.
Enasidenib is a first-in-class, oral, targeted inhibitor of mutant IDH2. The NDA submission is based on results from AG221-C-001, a single-arm phase I/II study of enasidenib in patients with advanced hematologic malignancies with an IDH2 mutation.
Enasidenib is also being evaluated compared with conventional therapy in older patients with an IDH2 mutation and relapsed or refractory AML in the ongoing phase III IDHENTIFY trial (NCT02577406). Enasidenib is an investigational drug that has not been approved for any use in any country.