Celgene, Agios beat a short path to an FDA decision on AML drug

David Schenkein

Celgene and Agios are flooring the gas pedal in their race to gain a very quick — and early — approval for enasidenib (AG-221/CC-90007), their experimental therapy for acute myeloid leukemia. Not only did the FDA accept their application, regulators are providing the inside track to a new approval with a priority review date that sets the PDUFA date at the end of August.

Agios $AGIO detailed its plans for taking a direct shot at an early approval last fall, earning a spike from investors as the biotech spotlighted the potential for taking Phase I/II study data to regulators. Celgene reworked its deal with Agios last year, but kept its hands on its rights to AG-221 in patients with advanced hematologic malignancies with an IDH2 mutation.

This is Agios’ lead drug, and it arrived at the FDA swiftly, by biotech standards. But CEO David Schenkein doesn’t think about drug development in traditional terms.

If you go to Agios’ website, you’ll see that the pipeline isn’t arranged according to preclinical, Phase I, Phase II and Phase III development.

Michael Pehl, Celgene

Instead, the biotech just divides the work into early and late stage development.

“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,” said Michael Pehl, president, Hematology/Oncology for Celgene, in a prepared statement. “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.”

Shares of Agios were up 3% in early trading Wednesday.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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