FDA Releases Six Draft Guidance Documents for Gene Therapy

Posted in :: 2018 Volume 3 :: Thursday, August 23rd, 2018

The United States Food and Drug Administration (FDA) released six draft guidance documents related to gene therapy. Three of the guidance documents are disease-specific, and three are related to manufacturing. According to Scott Gottlieb, MD, FDA Commissioner, these documents are the building blocks of the FDA’s framework for advancing gene therapy while requiring safety and effectiveness. The guidance is part of the FDA’s efforts to provide clear recommendations to sponsors and researchers.

Read FDA Commissioner’s Statements

Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry

The FDA highlights the importance of establishing standardized procedures for cell collection and handling across all collection sites for multi-center clinical trials to assure the quality and safety of the final product as well as ensuring control of the manufacturing process. A list of collection sites, the FDA Establishment Identifier, and accreditations for compliance with established standards (e.g., Foundation for the Accreditation of Cellular Therapy) should be included in the IND.

Additional Manufacturing Draft Guidance Documents

Long Term Follow-up After Administration of Human Gene Therapy Products; Draft Guidance for Industry

Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry

Disease-Specific Draft Guidance Documents

Human Gene Therapy for Hemophilia; Draft Guidance for Industry

Human Gene Therapy for Rare Diseases; Draft Guidance for Industry

Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry