Vertex Pharmaceuticals Says UK Approves Casgevy for Children Aged 5 and Older

06:08 AM EDT, 10/30/2026 (MT Newswires) -- Vertex Pharmaceuticals (VRTX) received approval from the UK's Medicines and Healthcare products Regulatory Agency, or MHRA, to use Casgevy for children aged five and older with severe sickle-cell disease and transfusion-dependent beta-thalassemia, according to a Friday statement from the regulator. The gene-editing treatment was previously authorized for patients aged 12 and above, with the treatment modifying bone marrow stem cells to enable the production of functioning haemoglobin, it said. The MHRA said the approval followed an assessment of the treatment's safety, quality and effectiveness, and that it would continue monitoring it. Reported side effects include nausea, fatigue, fever and an increased risk of infection, it added. Vertex is the exclusive license holder, manufacturer and commercial distributor of CASGEVY, the statement said.