Published date:
27/01/2023
Summary:
– Application is supported by results from two global Phase 3 studies of exa-cel in
people with sickle cell disease or transfusion-dependent beta thalassaemia –
London, UK and ZUG, Switzerland, January 27, 2023 – Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics (NASDAQ: CRSP) today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has validated the Marketing Authorisation Application (MAA) of exa-cel for the treatment of sickle cell disease (SCD) and transfusiondependent beta thalassaemia (TDT) in Great Britain. The submission is supported by two global Phase 3 studies investigating exa-cel as a potential one-time therapy for people with SCD or TDT.
