Vertex Sickle Cell Therapy Gets Second FDA Approval

The FDA has approved the Vertex Pharmaceuticals CRISPR/Cas9 gene-edited cell therapy called Casgevy for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of Casgevy for sickle cell disease [SCD], it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, CEO and President of Vertex.

TDT is a serious, life-threatening genetic disease that requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In the U.S. the lifetime health care costs of managing TDT are estimated to be between $5 and $5.7 million with a median age of death for patients of 37 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of patients because of the lack of available donors.

The administration of Casgevy requires experience in stem cell transplantation; therefore, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer Casgevy to patients.

Casgevy is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. (Source: Vertex Website, 16 January, 2024)