Enasidenib Granted Priority Review For Relapsed or Refractory Acute Myeloid Leukemia

The FDA accepted Celgene’s New Drug Application (NDA) for enasidenib (AG-221/CC-90007) and granted Priority Review for the treatment of relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation.

Enasidenib is a first-in-class, oral, targeted inhibitor mutant IDH2. The drug was given a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2017, according to a press release.

“We accelerated this application—–submitting the NDA just 3 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,” Michael Pehl, president of Hematology/Oncology for Celgene, said in the release. “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.”

The NDA submission—–completed by Celgene in late December 2016––is based on results from a single-arm, phase 1/2 study AG221-C-001, which examined enasidenib in patients with advanced hematologic malignancies with an IDH2 mutation.

Early data from patients with relapsed or refractory AML in the study were presented at the 2015 American Society of Hematology annual meeting, according to the release.

“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,” said David Schenkein, MD, chief executive officer at Agios. “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.”

Currently, enasidenib is being evaluated in the ongoing phase 3 IDHENTIFY trial, which compares the treatment with conventional therapy in older patients with an IDH2 mutation and relapsed or refractory AML

AML is a cancer of blood and bone marrow characterized by rapid disease progression. Among adults, AML is the most common acute leukemia, with a 5-year survival rate of approximately 20% to 25%. IDH2 mutations are present in approximately 8% to 19% of AML cases.