Regulatory Approval
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News
EU APPROVES MITAPIVAT | A New Treatment Milestone for Adults with α- or β-Thalassaemia
This decision is of particular importance because it covers a broad adult thalassaemia population, including both alpha- and beta-thalassaemia and both transfusion-dependent and non-transfusion-dependent disease. According to Agios Pharmaceuticals’ official…
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News
ACCESS TO INNOVATION | UK Greenlights Gene-Editing Therapy Casgevy for β-Thalassaemia Treatment
This development opens new treatment avenues for an estimated 460 patients aged 12 and above in England who rely on regular transfusions due to β-thalassaemia, leveraging innovative CRISPR gene-editing technology.…
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Scientific News
CASGEVY | 1st Gene-Editing Therapy for Thalassaemia and SCD Approved in UK
Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The therapy, developed by…
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