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# Enzyme Replacement Therapy Guidance
- URL: https://www.fdaweb.com/enzyme-replacement-therapy-guidance/
- Published: 2019-10-02T12:00:00.000Z
- Updated: 2026-09-15T01:53:54.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145148

FDA has published a guidance, *Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment*, to help sponsors develop and conduct nonclinical studies during development of investigational enzyme replacement therapy (ERT) products. Specifically, the [document](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/investigational-enzyme-replacement-therapy-products-nonclinical-assessment?ref=fdaweb.com) describes the agency’s current thinking about the substance and scope of nonclinical information needed to support start of clinical trials, ongoing clinical development, and marketing approval for investigational ERT products.

The guidance is intended as an adjunct to three International Council on Harmonization guidances that give general recommendations about the nonclinical safety studies of traditional small molecule and biotechnology-derived pharmaceuticals that support human clinical trials, as well as marketing authorization for pharmaceuticals.

The FDA guidance recommendations apply to ERT products indicated for lysosomal storage diseases and other diseases caused by inborn errors of metabolism. It does not apply to the development of pancreatic enzyme products.