beta-thalassemia
NICE Recommends Vertex's Casgevy for Beta Thalassemia Through Innovative Medicines Fund
The agency is making the drug available through the IMF so that more data can be collected on its efficacy and cost-effectiveness.
CHMP Recommends Conditional Approval for Vertex's Exa-cel in the EU
The EC will review the available data and decide by February 2024 whether to approve the CRISPR gene-edited therapy for sickle cell disease or beta thalassemia.