Vertex Pharmaceuticals and CRISPR Therapeutics have secured a label expansion for their gene editing therapy Casgevy (exagamglogene autotemcel) from UK regulators – further broadening the drug’s use case across the haematology treatment landscape.
Through this newfound approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), healthcare providers can now use Casgevy to treat patients with severe sickle cell disease and transfusion-dependent β-thalassemia aged five and up. This marks a notable expansion in the drug’s UK-based total addressable population, as it was previously only indicated for use in patients aged 12 and up with these conditions.
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While the National Institute for Health and Care Excellence (NICE) greenlit Casgevy’s use in patients aged 12 and up with both β-thalassemia and sickle cell disease in 2024 and 2025, respectively, it is currently unclear what stance the UK health watchdog will take on its use in patients aged between five and 12.
Vertex and CRISPR designed Casgevy, a non-viral gene therapy, to target the BCL11A gene to reactivate the production of an oxygen-carrying protein called foetal haemoglobin (HbF) – thus preventing red blood cells from forming their characteristic banana-like shape in sickle cell disease, while improving the functionality of red blood cells in β-thalassemia.
Casgevy first debuted on global market in 2023 after securing the regulatory greenlight from the US Food and Drug Administration (FDA) for the treatment of sickle cell disease, with the genetic medicine then netting a second US approval in β-thalassemia in 2024.
Most recently, the drug was approved by the FDA for use in patients aged two and above – a feat achieved just 53 days post-filing, according to Vertex.
“Severe sickle cell disease and β-thalassemia are serious, lifelong conditions that in some cases can be fatal,” noted the MHRA’s executive director of operations, Julian Beach, adding that the extension of Casgevy’s utility into a younger patient population was based on “careful assessment of the available evidence” around the therapy’s safety and efficacy.
Casgevy’s UK label expansion comes as the gene editing therapy continues to establish itself as a potential big seller for Vertex, as it posted revenue growth of 78% and 151% from a quarterly and yearly perspective in Q2 2026. Currently, analysts at GlobalData, parent company of Pharmaceutical Technology, forecast that Casgevy will have a blockbuster future, as they expect the drug to pull in sales of $1.6bn in 2032.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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