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
This document is intended to provide guidance to sponsors on the evidence necessary to 19 demonstrate the effectiveness of new drugs2 or new drug uses intended for slowly progressive, 20 low-prevalence rare diseases3 that are associated with substrate deposition and are caused by 21 single enzyme defects. This guidance applies only to those low-prevalence rare diseases with 22 well-characterized pathophysiology and in which changes in substrate deposition can be readily 23 measured in relevant tissue(s).
Submit comments on this guidance document electronically via docket ID: FDA-2013-S-0610 - Specific Electronic Submissions Intended For FDA's Dockets Management Staff (i.e., Citizen Petitions, Draft Proposed Guidance Documents, Variances, and other administrative record submissions)
If unable to submit comments online, please mail written comments to:
Division of Dockets Management (HFA- 305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852
All comments should be identified with the title of the guidance.