On January 16, the FDA approved Casgevy from Vertex, a cell-based gene therapy, for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassemia (TDT), an inherited disorder characterized by life-long anemia requiring frequent blood transfusions. To produce Casgevy, patients’ hematopoietic (blood) stem cells are modified by genome editing utilizing novel CRISPR/Cas9 technology. The modified blood stem cells are transplanted back into the patient where they engraft within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery and decreases the severity of anemia.

This is the second FDA-approved indication for Casgevy, which was approved in December for the treatment of sickle cell disease in patients 12 years and older. The current indication application received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.

“Today’s approval is an important step in the advancement of an additional treatment option for individuals with beta-thalassemia, a debilitating disease that places individuals at risk of many serious health problems,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “The approval of a cell-based gene therapy for this condition using CRISPR/Cas9 technology reflects FDA’s continued commitment to supporting safe and effective treatments that leverage the most promising and cutting-edge medical technologies.”

“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., CEO and President of Vertex. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible patients who are waiting.”