Agios gains FDA Priority Review for mitapivat in sickle cell disease
Agios Pharmaceuticals Inc. secured FDA Priority Review for its mitapivat sNDA targeting sickle cell disease, with a PDUFA goal date of November 1. The designation reduces the review timeline to six months. The application is supported by data from the RISE UP Phase 2 and Phase 3 trials.
*this image is generated using AI for illustrative purposes only.
Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with a Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date for this sNDA is November 1. This regulatory milestone targets a treatment for a serious condition with a potentially significant improvement in safety or efficacy. Following the announcement, the stock jumped by more than 15%.
The FDA’s Priority Review designation shortens the target review timeline from the standard 10 months to six months. The sNDA was submitted under the FDA’s accelerated approval pathway. Mitapivat is an oral pyruvate kinase (PK) activator.
RISE UP Trial Data
The application is based on data from the global, randomized, double-blind, placebo-controlled RISE UP Phase 2 and Phase 3 trials. These trials involved patients aged 16 years or older with sickle cell disease.
Current Approvals
Mitapivat is currently approved in the U.S. for adult patients in two hemolytic anemias. The drug received approval for PK deficiency in 2022 and for thalassemia in 2025.
How might FDA approval impact the competitive landscape of sickle cell disease treatments?
What are the potential market size and revenue projections for mitapivat in sickle cell disease?
Could the accelerated approval pathway influence future regulatory strategies for Agios?
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