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GUIDANCE DOCUMENT

Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment Guidance for Industry October 2019

Download the Final Guidance Document Read the Federal Register Notice
Final
Docket Number:
FDA-2015-D-1246
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research

The purpose of this guidance is to help sponsors design and conduct nonclinical studies during development of investigational enzyme replacement therapy (ERT) products. Specifically, this guidance describes the Food and Drug Administration’s (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.

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All written comments should be identified with this document's docket number: FDA-2015-D-1246.

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