Vertex Pharmaceuticals reports CHMP opinion for conditional approval of Casdevy

Vertex Pharmaceuticals announced that the European Medicines Agency’s, or EMA’s, Committee for Medicinal Products for Human Use, or CHMP, has adopted an opinion for the conditional approval of Casdevy, a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease, or SCD, and transfusion-dependent beta thalassemia, or TDT. If approved, exa-cel would be the only genetic therapy for patients in the European Union who are 12 years of age and older with either severe SCD with recurrent vaso-occlusive crises, or VOCs, or TDT, for whom hematopoietic stem cell, or HSC, transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available. An approval decision by the European Commission is expected in February 2024.