S12 Nonclinical Biodistribution Considerations for Gene Therapy Products; International Council for Harmonisation; Draft Guidance for Industry; Availability

Issuing agencies

Abstract

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "S12 Nonclinical Biodistribution Considerations for Gene Therapy Products." The draft guidance was prepared under the auspices of the International Council for Harmonisation (ICH), formerly the International Conference on Harmonisation. The draft guidance provides harmonized recommendations for the conduct and overall design of nonclinical biodistribution (BD) studies for gene therapy (GT) products. Considerations for interpretation and application of the BD data to support a nonclinical development program and inform the design of clinical trials are also provided. The recommendations in the guidance endeavour to facilitate the development of investigational GT products, while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles. The draft guidance is intended to promote harmonization of recommendations for BD studies for investigational GT products and facilitate a more efficient and timely nonclinical development program.