CRISPR Regulatory Updates

European Medicines Agency

European Medicines AgencyOn December 15, 2023, Vertex announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion for the conditional approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The news comes shortly after FDA approval of CASGEVY, which we recently reported here.

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 (VOCs) or TDT, for whom hematopoietic stem cell 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.

In related news, Charles River announced on December 18, 2023 that their Memphis facility was approved to manufacture Vertex’s CASGEVY. According to Charles River, the approval “follows Charles River’s Memphis center of excellence passing back-to-back audits from both the U.S. Food and Drug Administration (FDA) and the Health Products Regulatory Authority (HPRA), on behalf of the European Medicines Agency (EMA). The Memphis facility was the first North American contract development and manufacturing organization (CDMO) to be approved by the EMA to commercially manufacture an allogeneic cell therapy drug product.”