US FDA Nods Vertex's Casgevy To Treat Transfusion-dependent Beta Thalassemia

Vertex Pharmaceuticals Incorporated has announced that Casgevy, a CRISPR/Cas9 gene-edited cell therapy, has been approved by the US Food and Drug Administration (FDA) for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years of age and older.

“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., chief executive officer 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.”

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 US to offer Casgevy to patients. All nine ATCs activated in the US are able to offer Casgevy to eligible patients with TDT and sickle cell disease (SCD), as per pharmabiz.

TDT is a grave, hereditary condition that can be fatal. Patients with TDT have health-related quality of life scores that are lower than those of the general population, and the lifetime medical expenses associated with treating TDT are projected to be between $5 and $5.7 million in the US. Iron chelation therapy and regular blood transfusions are necessary for TDT patients for the duration of their lives. Patients with TDT may have anemia-related exhaustion and dyspnea, and newborns may experience jaundice, feeding issues, and failure to thrive. 

A swollen spleen, liver, or heart, malformed bones, and delayed puberty are other complications associated with TDT. Reduced life expectancy, a lower quality of life, lower lifetime earnings, and decreased productivity are the ultimate consequences of TDT, which necessitates lifelong treatment and a large investment of health care resources.