EMA — authorised 9 February 2024
- Application: EMEA/H/C/005763
- Marketing authorisation holder: Vertex Pharmaceuticals (Ireland) Limited
- Local brand name: Casgevy
- Indication: β thalassemia Casgevy is indicated for the treatment of transfusion dependent β thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available. Sickle cell disease Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is
- Pathway: conditional, orphan, ATMP, PRIME
- Status: approved
The European Medicines Agency (EMA) has approved Casgevy for the treatment of two rare blood disorders: transfusion-dependent β thalassemia and severe sickle cell disease. Casgevy is indicated for patients aged 12 and older who are not suitable for haematopoietic stem cell transplantation due to a lack of a matched related donor. This approval was granted under the conditional, orphan, advanced therapy medicinal product (ATMP), and PRIME expedited pathways.