November 16, 2023
UK Grants First-Ever Approval for CRISPR/Cas9 Gene-Edited Therapy, CASGEVY, in Sickle Cell Disease and Thalassemia Treatment
Vertex Pharmaceuticals and CRISPR Therapeutics have received conditional marketing authorization from the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) for Casgevy (exagamglogene autotemcel), marking the world's first regulatory approval for a CRISPR-based gene-editing therapy. Casgevy is designed for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
This groundbreaking therapy is indicated for eligible patients aged 12 and above with SCD, experiencing recurrent vaso-occlusive crises (VOCs), or TDT when a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is unavailable. It is estimated that around 2,000 patients in the U.K. may be eligible for Casgevy.
Global clinical trials of Casgevy in both SCD and TDT have demonstrated success, meeting their primary outcomes of achieving freedom from severe VOCs or becoming transfusion-independent for at least 12 consecutive months. These benefits are anticipated to be enduring once attained.
The safety profile, based on 97 patients with SCD and TDT treated with CASGEVY, aligns with myeloablative conditioning using busulfan and hematopoietic stem cell transplant.
In the U.K., exa-cel received an Innovation Passport through the Innovative Licensing and Access Pathway (ILAP) from the MHRA. Vertex is actively collaborating with national health authorities to facilitate swift access for eligible patients.