Casgevy is the first CRISPR-based medicine to be approved for use in the United States<\/p>\n<\/h3>\n
By Physician\u2019s Briefing Staff HealthDay Reporter<\/b><\/p>\n
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WEDNESDAY, Jan. 17, 2024 (HealthDay News) — After approving Casgevy (exagamglogene autotemcel)<\/a> in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat patients older than 12 years with transfusion-dependent beta-thalassemia.<\/p>\n Casgevy is the first CRISPR-based medicine, where gene editing is used to develop the treatment, to be approved for use in the United States. The one-time dose permanently changes DNA in a patient’s blood cells, but experts note the relief will not come cheap. The treatment list price is $2.2 million for its use in both sickle cell disease and beta-thalassemia, CNN<\/em> reported.<\/p>\n “Today’s approval is an important step in the advancement of an additional treatment option for individuals with beta-thalassemia,” Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA Center for Biologics Evaluation and Research, said in an agency news release<\/a>. “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.”<\/p>\n The latest FDA decision was expected, but it comes about two months earlier than the agency’s deadline for acting, CNN<\/em> reported.<\/p>\n To make Casgevy, a person’s stem cells are removed and modified using a gene-editing technique called CRISPR\/Cas9. The altered cells are then transplanted back into the patient’s body, where they multiply and increase the production of hemoglobin, which eases symptoms.<\/p>\n The most common side effects with Casgevy were mouth sores, febrile neutropenia, and decreased appetite, according to the FDA.<\/p>\n