This approval allows the treatment of children aged 2 years and older who suffer from sickle cell disease (SCD) with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT).
Sickle cell disease is a rare, inherited blood disorder that leads to the deformation of red blood cells, restricting blood flow and oxygen delivery to vital organs.
CASGEVY is the first genetic therapy approved for children as young as 2 years for both SCD and TDT, making approximately 5,500 additional children in the U.S. eligible for this one-time treatment.
Vertex is also pursuing regulatory reviews for label expansion in the Kingdom of Saudi Arabia and the United Kingdom.
Reshma Kewalramani, M.D., Chief Executive Officer and President, said, “Just as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia. The remarkable consistency of results across age groups reinforces the potential of CASGEVY to deliver durable, transformative benefits to those who have historically had limited options.”
