Now It Really Is Personal: US FDA 'Rushing' To Develop Policy For 'Bespoke Therapies'
Recent cases of individual designed treatments have FDA playing a bit of catch up. The concept has been on the horizon, but now “bespoke therapies” are here.
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Agency drafts guidance on how to apply requirements for chemistry, manufacturing and controls, and for good manufacturing practices, to bespoke antisense nucleotides.
US FDA’ s latest ‘n of 1’ guidance describes clinical considerations for INDs to support dosing and monitoring of an individual with a life-threatening genetic disease who is eligible to receive an ASO; recommendations apply only when a few patients may be candidates for treatment.
Draft guidance will help sponsor-investigators working on individualized gene therapies. The focus on basic administrative and procedural aspects of interaction with the FDA leaves many policy and commercialization questions about how these personalized investigational products should be studied unanswered.