Aggregated News

a sickle cell

The Food and Drug Administration on Tuesday approved Vertex Pharmaceuticals’ CRISPR-based medicine Casgevy for the inherited blood condition beta thalassemia, expanding its use six weeks after issuing a landmark clearance in sickle cell disease.

The agency’s decision makes Casgevy, which in December became the first CRISPR gene editing therapy to reach the U.S. market, available for people aged 12 years and older who have a severe form of beta thalassemia that requires regular blood transfusions. The regulator reached a verdict about two months ahead of schedule. 

Vertex said the same treatment centers it is working with to offer Casgevy to people with sickle cell will also be able to treat individuals with transfusion-dependent beta thalassemia, or TDT. So far, the company has authorized nine sites and expects to add more in the coming weeks. 

Casgevy will cost $2.2 million for TDT, the same price Vertex set for its use in sickle cell, a company spokesperson confirmed by email. About 1,000 people with TDT are estimated to be eligible for Casgevy in the U.S., far fewer than the number of eligible sickle...