EMA Authorised: Casgevy (Exagamglogene autotemcel) — Vertex Pharmaceuticals (Ireland) Limited

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Summary

EMA authorised Casgevy (exagamglogene autotemcel) by Vertex for beta-thalassemia and sickle cell disease, with conditional approval and orphan designation.

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Diseases / indications

• Beta-thalassemia
• Sickle Cell Disease

Drugs mentioned

• Casgevy (exagamglogene autotemcel)

Snippet

Status: Authorised. Therapeutic area: beta-Thalassemia;Anemia, Sickle Cell. MAH: Vertex Pharmaceuticals (Ireland) Limited. Decision date: 10/02/2026. Conditional approval. Orphan medicine