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NICE’s final draft guidance states that the one-time treatment will be made available on the NHS in England under a managed access scheme for patients aged 12 years and older who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.
Approximately 15,000 people in England are living with SCD, an inherited blood disorder which causes red blood cells to become irreversibly crescent shaped. This can block blood vessels and affect the way oxygen is carried around the body.
The disease can cause severe organ damage and intense pain, as well as haemolytic anaemia, a blood condition that occurs when red blood cells are destroyed faster than they are replaced.
Vertex’s Casgevy uses Nobel Prize-winning tool CRISPR to edit the faulty gene in patients’ bone marrow stem cells so that the body produces functioning haemoglobin. As patients receive their own edited cells, there is no risk of rejection.
In clinical trials, all patients who received the therapy avoided a hospitalisation for a year following treatment, and almost 98% had still avoided hospitalisation around 3.5 years later.
The list price of Casgevy is £1.65m, but NHS England outlined that it has secured a deal to access the treatment at a reduced price for taxpayers. It is estimated that around 50 patients will receive the therapy every year.
NHS chief executive, Amanda Pritchard, said: “More than a step, this is a leap in the right direction for people with SCD – which can be an extremely debilitating and painful condition.
“This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them.”
Sharing a similar sentiment, Ludovic Fenaux, senior vice president, Vertex International, said: “Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease.”