CASGEVY approved for use on the NHS in England
From 31 January 2025, NHS patients in England with sickle cell disease will have access to the one-time gene therapy, CASGEVY.
The National Institute for Health and Care Excellence (London, UK) has approved CASGEVY (exagamlogene autotemcel) for use on the NHS in England for patients who are aged 12 and older with a severe form of sickle cell disease.
Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England (London, UK), said:
“This represents a monumental step forward in the treatment of people with sickle cell disorder, which is a condition that mostly affects people of Black African and Black Caribbean heritage. This groundbreaking therapy, available on the NHS, represents a very real prospect of a cure for this devastating disorder.”
From the 31 January, CASGEVY will be offered at specialist NHS centers in London, Manchester and Birmingham. The treatment will be offered to patients who experience recurrent sickle cell crises and are suitable for a stem cell transplant but lack a suitable donor. It is estimated that around 50 patients could receive the treatment annually.
Through an agreement with Vertex Pharmaceuticals (MA, USA), NHS England has secured access to CASGEVY, originally priced at £1.65 million (US$2.04 million) per dose, at a reduced cost for taxpayers. This enables the treatment to be provided through the Innovative Medicines Fund, which fast-tracks funding for new treatments.
“The NHS remains committed to rolling out new, innovative treatments to narrow the gap in healthcare inequalities, and this cutting-edge therapy could give patients facing severe sickle cell disorder the hope of a much brighter future,” continued Owolabi.
More on CASGEVY
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Cell therapy weekly: FDA authorizes CASGEVY for beta thalassemia
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The CRISPR/Cas9 gene-edited therapy, CASGEVY, will be manufactured at Lonza’s (Basel, Switzerland) current Good Manufacturing Practice state-of-the-art facilities.
