pharmaphorum December 8, 2023
The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals’ Casgevy and bluebird bio’s Lyfgenia. The former is notably the first FDA-cleared gene therapy to utilise the CRISPR gene editing method.
FDA’s clearance of Casgevy follows a similar decision from the UK’s Medicines and Healthcare products Regulatory Agency less than a month ago. Both historic FDA approvals made use of a number of pathways created to fast track innovative treatments for rare diseases, receiving the Priority Review, Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy designations.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field...
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