pharmaphorum January 17, 2024
The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-dependent beta thalassaemia (TDT) more than two months ahead of deadline.
Casgevy (exagamglogene autotemcel or exa-cel) became the first drug based on the CRISPR/Cas9 gene-editing technology to be approved towards the end of last year, when it was cleared by the FDA for sickle cell disease (SCD). The UK was the first country to give the go-ahead for the drug last November, backing it for both TDT and SCD.
“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of...
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