FDA Approves Second Indication for the Gene Therapy Casgevy

FDA has approved Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited cell therapy, to treat transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. Beta thalassemia is a blood disorder that reduces the production of red blood cells. Patients report health-related quality of life scores below the general population and the lifetime healthcare costs in the United States are estimated between $5 and $5.7 million, according to a Vertex press release.

Casgevy was approved in early December 2023 to patients with sickle cell disease. It is a one-time gene therapy that uses a patient’s own hematopoietic stem cells that are edited to produce high levels of fetal hemoglobin in red blood cells. Patients receiving this therapy have stem cells extracted and then edited using CRISPR Therapeutics’ CRISPR/Cas9 technology. The edited stem cells are then infused back into the patient as part of an autologous hematopoietic stem cell transplant. So far, nine authorized treatment centers have been selected to administer the therapy.

Casgevy has a wholesale acquisition cost of $2.2 million. Vertex offers a program, called Vertex Connects, with care managers available to help providers and patients with educational resources, communications and support to navigate treatment journey.

Related: FDA Approves Two Gene Therapies for Sickle Cell Disease

Additionally, in December, the FDA approved a second gene therapy for sickle cell disease: Bluebird bio’s Lyfgenia (lovotibeglogene autotemcel or lovo-cel), which is a one-time treatment that is designed to add functional copies of a modified form of the β-globin gene into a patient’s own blood stem cells, enabling the body to produce its own hemoglobin. It does not use CRISPR technology.

Bluebird has set the wholesale acquisition cost of of Lyfgenia at $3.1 million. Executives said this price recognizes the value the therapy may deliver. Frequent vaso-occlusive events average between $4.0 and $6.0 million in direct lifetime medical costs, Bluebird said in a press release. Outcomes-based contract offerings available to both commercial payers and Medicaid.