Boston — Vertex Pharmaceuticals has received U.S. Food and Drug Administration approval to expand the use of CASGEVY to people ages 2 and older with severe sickle cell disease or transfusion-dependent beta thalassemia.
CASGEVY, also known as exagamglogene autotemcel, is now the first approved genetic therapy available to children as young as 2 for both sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta thalassemia.
The expanded approval makes approximately 5,500 additional children in the United States eligible for the one-time treatment. CASGEVY was previously approved for patients ages 12 and older.
“Just as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia. The remarkable consistency of results across age groups reinforces the potential of CASGEVY to deliver durable, transformative benefits to those who have historically had limited options,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We’re deeply grateful to the patients, families and investigators who participated in the clinical trials that led to this historic approval, and we are ready to bring CASGEVY to children and their families across the U.S.”
CASGEVY is a nonviral, ex vivo CRISPR/Cas9 gene-edited cell therapy. A patient’s own blood-forming stem and progenitor cells are edited at a region of the BCL11A gene to increase production of fetal hemoglobin.
Clinical trials have shown that the therapy can reduce or eliminate vaso-occlusive crises in people with sickle cell disease and reduce or eliminate the need for transfusions in patients with transfusion-dependent beta thalassemia.
“Today’s approval offers renewed hope for children living with sickle cell disease or transfusion-dependent beta thalassemia,” said Haydar Frangoul, M.D., M.S., Medical Director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, investigator with Sarah Cannon Research Institute (SCRI) and Member of Vertex’s SCD Program Steering Committee. “Earlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold.”
Vertex has established more than 75 authorized treatment centers across the United States to provide CASGEVY to eligible patients through existing reimbursement and access pathways.
The FDA approved the expanded indication through the Commissioner’s National Priority Voucher program. Vertex has also submitted applications in Saudi Arabia and the United Kingdom to expand CASGEVY’s use to children as young as 5.
The approval was supported by the CLIMB clinical trial program. The completed Phase 1/2/3 CLIMB-111 and CLIMB-121 studies evaluated a single dose of CASGEVY in patients ages 12 to 35 with transfusion-dependent beta thalassemia or sickle cell disease with recurrent vaso-occlusive crises.
The ongoing Phase 3 CLIMB-141 and CLIMB-151 studies are evaluating the therapy in children ages 2 to 11. Enrollment and dosing have been completed for the 5-to-11-year-old groups in both studies.
Patients are also being asked to participate in CLIMB-131, a long-term study evaluating CASGEVY’s safety and efficacy for as long as 15 years following treatment.
