On February 9, 2024, the European Medicines Agency (EMA) issued a marketing authorisation for eExagamglogene autotemcel (Casgevy, by Vertex) to treat severe sickle cell disease.
Severe sickle cell disease (SCD) is a hereditary blood disorder marked by the production of abnormal haemoglobin, specifically haemoglobin S. This genetic anomaly leads to the distortion of red blood cells into a characteristic sickle or crescent shape, which significantly impairs their ability to transport oxygen efficiently throughout the body. The irregular shape of these cells causes them to become rigid and sticky, resulting in blockages within small blood vessels.
This can trigger a cascade of complications, including acute and chronic pain crises, caused by restricted blood flow, as well as chronic anaemia. Individuals with severe sickle cell disease are also at a heightened risk of infections requiring vigilant monitoring and preventive measures, such as vaccinations and prophylactic antibiotics.
If left untreated, severe sickle cell disease can lead to significant morbidity, manifesting in various forms such as organ damage, stroke, and pulmonary complications, ultimately resulting in a diminished quality of life for affected individuals.
On the 26th of February this year, NICE published its technology appraisal guidance on Casgevy, recommending it for use with a managed access program for patients who are 12 years old and over, who have recurrent vaso-occlusive crises (VOCs) and a specific genotype, and when haematopoietic stem cell transplant is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available. It is only recommended for people who have had at least 2 VOCs per year during the previous 2 years and if the conditions in the managed access agreement are followed. The list price for a single dose of Casgevy is just over £1.6 million, the commercial access agreement makes it accessible to the NHS.
This innovative approach aims to provide critical treatment options for patients facing severe complications from their condition, ensuring they receive the necessary care even in the absence of a suitable donor.
