FDA Expands Use of First Sickle Cell Disease Gene Therapy for Children as Young as 2 Years

WEDNESDAY, July 8, 2026 (HealthDay News) -- The U.S. Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). 

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U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease

CAMBRIDGE, Mass., July 07, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease with a Priority Review.

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African health leaders and partners launch new framework to strengthen sickle cell disease care across the continent

NAIROBI, Kenya / KAMPALA, Uganda  — African health leaders and partners today launched the Imara Sickle Cell Framework, a new regional initiative designed to help countries strengthen sickle cell disease care by translating policy priorities into practical, coordinated delivery within national health systems.

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