The Road to FDA Approval for Gene Therapy
The FDA’s focus is on QUALITY, EFFICACY, and SAFETY. In support of these standards, the FDA provides guidelines for the preclinical development and evaluation of gene therapy products. The FDA states that gene therapy products “mediate their effects by transcription and/or translation of transferred genetic material and/or by integrating into the host genome and are administered as nucleic acids, viruses, or genetically engineered microorganisms.” Although only four approvals have been granted to date, there are more than 900 investigational new drug (IND) applications for gene therapy products in the pipeline.
Gene therapy products are biological products regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies in humans require the submission of an investigational new drug application (IND) before initiating clinical studies in the United States. Marketing a gene therapy product requires submission and approval of a biologics license application (BLA).