Investigational Enzyme Replacement Therapy (ERT) Products: Nonclinical Assessment

On October 1, 2019 FDA published this guidance for comment purposes only. Submit comments by January 2, 2020.

This guidance provides consistent recommendations for nonclinical studies to expedite developments of ERT products used to treat these rare, life-threatening conditions, especially in pediatric patients.

The purpose of this guidance is to help sponsors design and conduct nonclinical studies during development of investigational ERT products. Specifically, this guidance describes the FDA’s current thinking about the substance and scope of nonclinical information needed to support initiation of clinical trials, ongoing clinical development, and marketing approval for investigational ERT products.

This guidance is intended as an adjunct to the ICH guidances for industry M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals (January 2010), M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals — Questions and Answers (February 2013), and S6(R1) Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals (May 2012).

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