Agios Targets Accelerated Approval for Mitapivat in Sickle Cell Disease

Agios Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of mitapivat for sickle cell disease (SCD), advancing the regulatory process following the company’s previously announced plans for submission.

As part of the accelerated approval pathway, Agios and the FDA have aligned on the design of a required confirmatory clinical trial intended to verify the clinical benefit of mitapivat in reducing transfusion burden among patients with sickle cell disease.

The planned global study will be a randomized, double-blind, placebo-controlled 52-week trial enrolling approximately 159 patients aged 12 years and older with sickle cell disease. The primary endpoint will assess the proportion of patients remaining transfusion-free from Week 4 through Week 52.

According to the company, the confirmatory study design was informed by reductions in transfusion burden observed with mitapivat in the Phase 3 RISE UP trial based on prospectively collected data. Additional findings from the RISE UP study are scheduled for presentation during the Plenary Abstracts Session at the 31st European Hematology Association (EHA) Congress on June 13, 2026, in Stockholm, Sweden.

Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios, said the submission represents an important milestone for the sickle cell disease community and highlighted the company’s ongoing engagement with the FDA during the review process.

Agios stated that it expects to receive notice regarding filing acceptance and the anticipated FDA review timeline in the third quarter of 2026 following completion of the agency’s 60-day filing review period.

Previously, the U.S. FDA approved AQVESME as the first treatment for anemia in adults with alpha- or beta-thalassemia.

Reference

Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease – Agios Pharmaceuticals, Inc.