Published date:
25/01/2023
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 thalassemia –
LONDON, UK and ZUG, Switzerland, January 25, 2023 – Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application of exa-cel for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). 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.
