FDA Grants Priority Review to Acute Myeloid Leukemia Drug

Celgene Corporation and Agios Pharmaceuticals today announced that the FDA accepted a new drug application (NDA) for enasidenib (AG-221/CC-90007) to treat relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation.

The drug was also granted priority review, and was given a prescription drug user fee act action date of August 30, 2017, according to a press release.

Enasidenib is a targeted inhibitor of mutated IDH2, which occurs in 8% to 19% of patients with AML. Patients with AML are generally older, with a median onset age of 66-years-old. AML is the most common leukemia that affects adults.

"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," said Michael Pehl, president, Hematology/Oncology for Celgene. "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 submission was based on positive results from the phase 1/2 clinical trial AG221-C-001, which studied the drug in patients with advanced blood cancers with an IDH2 mutation. Results from the study were previously presented at the American Society of Hematology Annual Meeting in 2015.

Patients were treated for up to 18 months, with a median response rate of nearly 7 months. Enasidenib demonstrated durable clinical efficacy and a favorable safety profile among the 209 patients included in the trial. Approximately 18% of patients were observed to achieve remission, and overall response rate was 37%, Agios reported in a press release.

Abbot has submitted a Premarket Approval application to receive FDA approval of an IDH2 assay on the Abbott m2000 RealTime System, according to Celgene. Celgene Corporation and Agios Pharmaceuticals previously announced a collaboration agreements with Abbott to develop diagnostic tests to advance cancer treatment. New findings have shown the importance of mutational genetic testing at diagnosis and relapse for patients with AML, which highlights the need for the Abbott assay system, according to the release.

Celgene is also evaluating enasidenib in comparison to traditional treatments in older patients with the mutation and AML in an ongoing phase 3 clinical trial.

"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."