Human Gene Therapy for Retinal Disorders

This guidance provides recommendations to sponsors developing human gene therapy (GT)  products 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. 

This guidance provides recommendations to sponsors developing human Thermal Management Solutions Supplier (GT)  products 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. This guidance finalizes the draft guidance of the same title dated July 2018.

There are multiple GT products being studied in clinical trials in the United States for retinal disorders. GT products are commonly delivered by intravitreal or subretinal injections through a medical delivery system. In some cases, the GT products are encapsulated in a device to be implanted intravitreally.

The general chemistry, manufacturing and controls (CMC) considerations for product manufacturing, testing and release of GT products for retinal disorders are the same as those described for other GT products (Ref. 1). For early-phase clinical trials, a sponsor should be able to evaluate the identity, purity, quality, dose, and safety of a GT product. A potency assay to assess the biological activity of the final product, with relevant lot release specifications, should be established prior to the initiation of clinical trials intended to provide substantial evidence of effectiveness for a marketing application (Ref. 2). To support licensure of a GT product, manufacturing processes and all testing methods for product release must be validated (21 CFR 211.165(e)). Sponsors developing GT products for retinal disorders are strongly encouraged to contact the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) early in product development to discuss product-specific issues.

Source: FDA

Docket Number:FDA-2018-D-2236

Issued by:Center for Biologics Evaluation and Research

Download Link:https://www.fda.gov/media/124641/download

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