In a groundbreaking development, Vertex Pharmaceuticals and CRISPR Therapeutics have announced that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Casgevy, a CRISPR/Cas9 gene editing therapy. This marks the world’s first regulatory approval of a CRISPR-based therapy. Casgevy is designed for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Casgevy has received authorization for treating patients aged 12 and older with SCD experiencing recurrent vaso-occlusive crises (VOCs) or TDT when a suitable human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is unavailable. Approximately 2,000 patients in the U.K. are estimated to be eligible for this treatment. Reshma Kewalramani, MD, CEO, and president of Vertex, expressed the significance of this achievement, calling it a historic day in science and medicine. Casgevy’s conditional approval is expected to provide a new option for eligible patients awaiting innovative therapies. Samarth Kulkarni, PhD, Chairman and CEO of CRISPR Therapeutics, emphasized the potential of this Nobel Prize-winning technology to benefit patients with serious diseases.
Clinical trials of Casgevy in both SCD and TDT globally have shown positive outcomes, with patients achieving freedom from severe VOCs or transfusion independence for at least 12 consecutive months. The therapy’s safety profile, based on 97 patients treated in ongoing studies, aligns with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.
Josu de la Fuente, principal investigator in the CLIMB-111 and CLIMB-121 studies, expressed anticipation for patients to access this therapy swiftly. SCD is an inherited blood disorder causing severe pain and organ damage due to misshapen red blood cells, with stem cell transplants as the current cure. Beta thalassemia, another target of Casgevy, is characterized by anemia and associated symptoms. Casgevy, a genetically modified autologous cell population, undergoes CRISPR/Cas9 editing to address the genetic causes of these conditions. This milestone reflects a major advancement in the field, offering hope for patients with limited treatment options.