Vertex's gene therapy Casgevy has been approved to treat children, expanding access to a treatment previously cleared for older patients, according to Endpoints News.
The therapy can now be used for either of two inherited blood disorders: sickle cell disease with recurrent vaso-occlusive crises, or transfusion-dependent β-thalassemia. Both are painful, lifelong conditions caused by faulty genes that affect how the body produces healthy red blood cells.
Endpoints News reports that the expanded approval extends Casgevy's use to younger patients than before, opening the door for children living with these diseases to receive the treatment.
Casgevy is notable as one of the first therapies to reach patients using gene-editing technology. Rather than managing symptoms with ongoing medication or transfusions, gene therapies like this aim to correct the underlying genetic problem, offering the possibility of a durable, one-time treatment.
The same Endpoints News report noted separately that United Therapeutics agreed to acquire a startup, though details of that deal are distinct from the Vertex approval.
Why it matters: Extending a cutting-edge gene therapy to children means families dealing with sickle cell disease and β-thalassemia may be able to intervene earlier in life, potentially before the diseases cause their most serious, cumulative damage.