Agios Pharmaceuticals is awaiting a crucial FDA decision on its supplemental New Drug Application (sNDA) for Pyrukynd (mitapivat), aimed at treating adult patients with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The FDA has granted a standard review and set a Prescription Drug User Fee Act (PDUFA) goal date of September 7, 2025.
If approved, Pyrukynd, a pyruvate kinase (PK) activator, would mark a significant advancement in thalassemia treatment, potentially becoming the first oral therapy available for all thalassemia subtypes. Thalassemia, a rare inherited blood disorder, leads to chronic anemia and can result in severe complications such as organ damage and stroke. Current treatment options are limited, highlighting the urgent need for effective therapies.
Clinical Trial Data Supporting sNDA
The sNDA is supported by data from two Phase 3 clinical trials:
- ENERGIZE: Evaluated Pyrukynd in adults with non-transfusion-dependent alpha- or beta-thalassemia.
- ENERGIZE-T: Assessed Pyrukynd in adults with transfusion-dependent alpha- or beta-thalassemia. Results were presented at the 66th American Society of Hematology Annual Meeting and Exposition (ASH 2024) in December 2024.
Both studies met their primary and key secondary endpoints, demonstrating a favorable benefit-risk profile. These findings underscore Pyrukynd's potential to improve hemoglobin levels and reduce transfusion burden in thalassemia patients, regardless of their genotype or transfusion needs.
Ongoing Clinical Development
Agios is also advancing mitapivat in other hematologic disorders. The Phase 3 RISE UP study, evaluating mitapivat in sickle cell disease patients aged 16 and older, has completed enrollment, with topline results expected in late 2025. A potential U.S. commercial launch for sickle cell disease is anticipated in 2026.
Financial Highlights and Corporate Strategy
In February 2025, Agios reported fourth-quarter and full-year 2024 financial results, highlighting Pyrukynd net revenue of $10.7 million for the quarter and a cash position of $1.5 billion as of December 31, 2024. This financial strength supports Agios' strategic priorities, including maximizing the potential of the Pyrukynd franchise and expanding its pipeline.
Brian Goff, CEO of Agios, stated, "Our PYRUKYND franchise is poised for multi-billion-dollar potential, driven by the key milestones we achieved last year, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our Phase 3 RISE UP study for sickle cell disease."
Safety Information
Pyrukynd carries warnings regarding acute hemolysis and hepatocellular injury. Abrupt discontinuation should be avoided, and liver tests should be monitored regularly. Common adverse reactions include decreased estrone (males), increased urate, back pain, decreased estradiol (males), and arthralgia.
