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GUIDANCE DOCUMENT

Human Gene Therapy for Retinal Disorders Draft Guidance for Industry July 2018

Draft

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Docket Number:
FDA-2018-D-2236
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

This guidance provides recommendations to stakeholders developing human gene therapy (GT) products1 for retinal disorders affecting adult and pediatric patients. These disorders vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. These disorders manifest with central or peripheral visual impairment and often with progressive visual loss. This guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products.


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-2236.

Questions?

Contact Point
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
ocod@fda.hhs.gov
(800) 835-4709
(240) 402-8010