UK – NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

The National Institute for Health and Care Excellence (NICE) has approved Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) gene therapy to treat a severe form of sickle cell disease (SCD).

NICE’s final draft guidance states that the one-time treatment will be made available on the NHS in England under a managed access scheme for patients aged 12 years and older who experience recurrent sickle cell crises.

Those eligible for the therapy will also be suitable for a stem cell transplant, but will not have a matched donor…