US FDA Limits GMP Exemption For ‘N of 1’ Investigational New Drugs
Agency drafts guidance on how to apply requirements for chemistry, manufacturing and controls, and for good manufacturing practices, to bespoke antisense nucleotides.
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The White House ‘Biotechnology’ initiative is very broadly focused, with non-health sectors receiving the bulk of the attention. The US FDA’s role is small – but it could further boost attention to efforts to encourage adoption of advanced manufacturing.
Draft guidance will help sponsor-investigators working on individualized gene therapies.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.
One Patient, Many Questions: Milasen ‘N of 1’ Trial Provides Early Template For Individualized Genomic Medicine
Rapid development of patient-customized oligonucleotide at Boston Children’s Hospital suggests promise and hazards of new drug development model.