US Guidance for Industry update

The U.S. Food and Drug Administration (FDA) has recently published the following draft and final Guidance for Industry documents of relevance to developers / manufacturers of cell- and gene-based therapies.

The draft Guidance for Industry document on MVGTs describes in detail how product manufacturing, nonclinical studies, and clinical studies using such vectors should be designed and executed for IND (Investigational New Drug) submissions. These products include those used both to modify cellsin vivo or to transfer to cells ex vivo prior to administration to the recipient. FDA invites comments on this draft guidance until 14 December 2015.

The Product Development Under the Animal Rule guidance provides information and recommendations on drug and biological product development when human efficacy studies are not ethical or feasible, meaning that approvals are based on animal studies alone. The regulations that set forth the pathway for approval of these products under 21 CFR 314.600 through 314.650 (drugs) or 21 CFR 601.90 through 601.95 (biological products) are commonly referred to as the Animal Rule. This finalised guidance document is structured with detailed sections on:

  • Regulatory Considerations
  • Animal Studies—General Expectations
  • Essential Elements of an Animal Model
  • Design Considerations for the Adequate and Well-Controlled Efficacy Studies in Animals
  • Considerations for Preventive Vaccines and for Cellular and Gene Therapies
  • Human Safety Information

Author: Anthony Lodge

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