{"id":614404,"date":"2024-06-14T10:28:48","date_gmt":"2024-06-14T14:28:48","guid":{"rendered":"https:\/\/platohealth.ai\/vertex-says-casgevy-benefit-extends-to-nearly-five-years-in-sickle-cell-disease\/"},"modified":"2024-06-14T13:52:53","modified_gmt":"2024-06-14T17:52:53","slug":"vertex-says-casgevy-benefit-extends-to-nearly-five-years-in-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/vertex-says-casgevy-benefit-extends-to-nearly-five-years-in-sickle-cell-disease\/","title":{"rendered":"Vertex says Casgevy benefit extends to nearly five years in sickle cell disease","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
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The data is from more than 100 patients \u2013 46 with sickle cell disease and 56 with TDT \u2013 treated with Casgevy. Image credit: Shutterstock \/ JHVEPhoto.<\/figcaption><\/figure>\n<\/p><\/div>\n
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Vertex Pharmaceuticals<\/a> has reported long-term data for its gene therapy Casgevy (exa-cel) in patients with sickle cell disease or transfusion-dependent beta-thalassemia (TDT).<\/p>\n

Currently boasting the only approved CRISPR-based gene editing therapy, Vertex presented the results from the long-term follow-up trials at the annual European Haematology Association (EHA) Congress, held in Madrid, Spain from 13 to 16 June.<\/p>\n

The data is from more than 100 patients \u2013 46 with sickle cell disease and 56 with TDT \u2013 treated with Casgevy.<\/p>\n

Casgevy was approved by the US Food and Drug Administration (FDA)<\/a> in December 2023 alongside bluebird bio<\/a>\u2019s Lyfgenia (lovo-cel). This was followed by a European approval<\/a> in February 2024.<\/p>\n

While gene therapies have the potential to transform the treatment<\/a> of rare diseases, questions remain over long-term effectiveness and safety<\/a>.<\/p>\n

The longer-term data from Vertex\u2019s CLIMB trials could go some way to quell any circling over Casgevy. In sickle cell disease, 92.3% of patients with at least 16 months of follow-up were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months, consolidating previously reported primary endpoint data. The longest VOC-free duration was 54.8 months, whilst the mean was 27.9 months. All but one of the patients in the evaluable cohort were free from hospitalisations related to VOCs for at least 12 consecutive months.<\/p>\n

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