Pharmaceutical Business review

FDA grants priority review to Enasidenib for IDH2-Mutant AML

The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of Aug. 30, 2017. Celgene completed the NDA submission in late December 2016.

Celgene Hematology/Oncology president Michael Pehl said: "We accelerated this application – submitting the NDA just three years after the first patient was treated in the enasidenib pivotal investigational trial – because we believe that there is a significant unmet need for people with relapsed or refractory AML.

"The acceptance of the enasidenib NDA is a significant milestone in what we hope will be a new era of molecularly targeted therapies for patients with this devastating disease."

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.

Early data from the relapsed or refractory AML patients in this study were presented at the 2015 American Society of Hematology (ASH) Annual Meeting.

Agios CEO David Schenkein said: "Having received NDA acceptance and priority review for enasidenib, we look forward to working with our partner Celgene and the FDA to advance a first-in-class therapy for relapsed or refractory AML with an IDH2 mutation.

"We hope that the continued adoption of molecular profiling and availability of new targeted therapies such as enasidenib will have a significant impact on patients living with AML."

Additionally, Abbott has submitted a Premarket Approval (PMA) application for the FDA approval of an IDH2 assay on the Abbott m2000 RealTime System, a polymerase chain reaction (PCR) molecular diagnostics instrument.

IDH2 mutations occur in about 8 to 19 percent of AML patients. Recent publications have highlighted the advances in the understanding of the genetics underlying AML and the need for routine mutational analysis at diagnosis and relapse.

Celgene is also evaluating enasidenib 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.