The Food and Drug Administration (FDA) has approved a second indication for a drug developed using a gene editing process called CRISPR.
Casgevy, initially authorised in December for sickle cell disease treatment, is a groundbreaking gene therapy that is now also sanctioned for treating transfusion-dependent beta-thalassemia.
Beta thalassemia, like sickle cell disease, is a hereditary blood disorder. The FDA's approval, arriving approximately two months ahead of the scheduled PDUFA date, marks a significant advancement in treating these blood disorders.
Casgevy involves a precision gene editing process using CRISPR/Cas9 to modify a patient's stem cells. These altered cells are then reintroduced into the patient's body, fostering the growth of cells that produce more haemoglobin, thereby reducing symptoms.
The treatment was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.