BRIEF—MHRA to consider gene therapy for sickle cell disease

The UK's Medicines and Healthcare products Regulatory Agency (MHRA) is to review a submission for exagamglogene autotemcel (exa-cel), from USA-based Vertex Pharmaceuticals and Swiss firm CRISPR Therapeutics.

The British marketing authorization application is for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassaemia (TDT).

The news comes shortly after it was  announced that the European Medicines Agency has also accepted and will review a regulatory submission for the one-time gene therapy.

Chief regulatory and quality officer Nia Tatsis said the latest regulatory news was “a significant milestone,” adding: “We look forward to working with the MHRA on our application, the first for a CRISPR-based therapy for a genetic disease.”

Vertex has requested Orphan Drug designation as part of the application.