On Thursday, the U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for Agios Pharmaceuticals, Inc.’s supplemental New Drug Application (sNDA) of Pyrukynd (mitapivat) for adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia by three months to December 7, 2025. 

Following a recent information request from the FDA, Agios submitted a proposed Risk Evaluation and Mitigation Strategy (REMS) to mitigate the risk of hepatocellular injury that was described in the original Pyrukynd sNDA.

The submission of the REMS is a significant amendment to the Pyrukynd sNDA, resulting in a three-month review extension. This extension is not the result of new or additional efficacy or safety data requested by the FDA or submitted by Agios.

Also Read: Agios Pharmaceuticals Reveals Encouraging Data From Rare Blood Disorder Study In Younger Patients Aged Below 18 Years

The Pyrukynd sNDA is supported by results from ENERGIZE and ENERGIZE-T Phase 3 trials in adults with NTD and TD alpha- or beta-thalassemia, respectively.

Last month, the Saudi Food and Drug Authority (SFDA) approved Pyrukynd for adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

In 2024, Agios entered into a distribution agreement with NewBridge Pharmaceuticals to advance regulatory filings and commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region, which includes Saudi Arabia, the United Arab Emirates, Kuwait, Qatar, Oman, and Bahrain. 

Pyrukynd is also approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the U.S., and for PK deficiency in adult patients in the European Union and Great Britain.

Price Action: AGIO stock is down 15.83% at $34.18 at the last check on Thursday.

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