Vertex Pharmaceuticals has received expanded approval from the U.S. Food and Drug Administration for CASGEVY® (exagamglogene autotemcel), allowing the gene-editing therapy to be used in patients aged 2 years and older with sickle cell disease or transfusion-dependent beta thalassemia.
The decision makes CASGEVY the first approved genetic therapy available for children as young as two years old with either sickle cell disease, also known as SCD, or transfusion-dependent beta thalassemia, or TDT. The expanded indication applies to patients with recurrent vaso-occlusive crises in sickle cell disease and patients who require regular blood transfusions because of beta thalassemia.
CASGEVY is a one-time cell-based gene-editing therapy designed to address the underlying biology of both inherited blood disorders. The treatment uses a patient’s own blood stem cells, which are collected and edited outside the body before being infused back into the patient following conditioning treatment.
Sickle cell disease is a group of inherited blood disorders that causes red blood cells to become rigid and sickle-shaped. These cells can block blood flow and trigger painful vaso-occlusive crises, which may lead to organ damage, hospitalizations and reduced life expectancy. Transfusion-dependent beta thalassemia is another inherited blood disorder in which the body cannot produce enough functional hemoglobin, often requiring patients to undergo lifelong blood transfusions.
The FDA’s expanded approval is expected to provide earlier access to CASGEVY for children who may otherwise face years of complications from these chronic and potentially life-threatening conditions. Earlier treatment could help reduce the cumulative health impact associated with repeated pain crises, transfusions and disease-related organ damage.
Vertex said the approval was supported by consistent clinical results across different age groups. The company described the decision as an important milestone in its effort to expand access to transformative therapies for people with serious genetic diseases.
Reshma Kewalramani, chief executive officer and president of Vertex, said the expanded approval reinforces the potential of CASGEVY to provide durable benefits for patients with sickle cell disease and beta thalassemia. She added that the company is preparing to make the therapy available to eligible children and families across the United States.
Haydar Frangoul, medical director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, said the approval could give clinicians and families an opportunity to consider gene-editing treatment before children experience years of cumulative disease-related damage.
Vertex has established a network of independently operated authorized treatment centers across the United States to provide CASGEVY to eligible patients. The company said access will be offered through existing reimbursement and patient-support pathways.
The expanded FDA approval marks another significant development for gene therapy and gene editing in rare blood disorders. As treatment centers gain experience with CASGEVY, the therapy could reshape care for younger patients with sickle cell disease and transfusion-dependent beta thalassemia who have historically faced limited long-term treatment options.