Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) have received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for their jointly developed therapy, Casgevy (exagamglogene autotemcel), a treatment for sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for transfusion-dependent beta thalassemia (TDT) in eligible patients unable to access stem cell transplant. This single-use treatment marks the world’s first CRISPR-based gene-editing therapy to gain regulatory authorization.
One-Year Renewable Approval and Clinical Benefits
The 1-year renewable approval is supported by interim results from two ongoing, separate Phase II/III studies in SCD or TDT, where patients experienced no severe VOCs or transfusions for at least 12 months, with the potential for lifelong benefits.
Casgevy’s CRISPR/Cas9 Technology and Its Impact
Casgevy is composed of human, autologous hematopoietic stem and progenitor cells edited in the enhancer region of the BCL11A gene using CRISPR/Cas9 technology, aiming to produce fetal hemoglobin in red blood cells.- Flcube.com
