Vertex, CRISPR Therapeutics Earn EU Priority Drug Status for Gene Therapy

Vertex Pharmaceuticals and CRISPR Therapeutics have received European Medicines Agency priority designation (PRIME) for CTX001, an investigational gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

The new status was supported by data from an ongoing phase 1/2 trial evaluating patients with TDT, the companies said. The treatment previously received PRIME designation for SCD and Orphan Drug designation for SCD and TDT.

The experimental gene therapy has also secured Fast-Track, Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations from the FDA for both SCD and TDT.