Vertex Pharmaceuticals and CRISPR Therapeutics's gene editing therapy exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease and beta-thalassemia has received authorization from the UK's Medicines and Healthcare Products Regulatory Agency for patients aged 12 and older.
Following the authorization, the drug is set to be initially available for up to 2,000 patients in the UK. The price of the treatment is yet to be disclosed.
Analyst QuickTake: Initiated as part of a collaboration between Vertex and CRISPR in 2015, Casgevy marks the first CRISPR-based gene editing treatment to receive market authorization for two blood disorders. The drug is also nearing its market entry in the US, with the FDA’s decision expected on December 8, 2023 (exa-cel for sickle cell disease patients) and March 30, 2024 (for beta-thalassemia patients). Just last month , exa-cel received positive remarks from the FDA’s advisory team indicating a potentially positive decision from the FDA.
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