Occasional Digest

Vertex gains expanded indication for Casgevy in children as young as two

Sickle cell anemia, blood vessel, human anatomy biology

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  • The US FDA has approved Vertex Pharmaceuticals’ (VRTX) gene therapy Casgevy (exagamglogene autotemcel) for children as young as two years old with sickle cell disease or β thalassemia.
  • The approval is the eighth under the Commissioner’s National Priority Voucher pilot

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