U.S. flag An official website of the United States government
  1. Home
  2. Regulatory Information
  3. Search for FDA Guidance Documents
  4. Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies Guidance for Industry
  1. Search for FDA Guidance Documents

GUIDANCE DOCUMENT

Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition That Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies Guidance for Industry Guidance for Industry March 2020

Final
Docket Number:
FDA-2018-D-2456
Issued by:
Guidance Issuing Office
Office of Medical Products and Tobacco, Center for Drug Evaluation and Research
Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research

This document provides guidance to sponsors on the evidence necessary to demonstrate the effectiveness of investigational new drugs or new drug uses intended for slowly progressive, low-prevalence rare diseases that are associated with substrate deposition and are caused by single enzyme defects. This guidance applies only to those low-prevalence rare diseases with well-characterized pathophysiology, and in which changes in substrate deposition can be readily measured in relevant tissue or tissues.


Submit Comments

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2018-D-2456.