FDA Releases New Draft Guidances on Gene Therapies

The FDA unveiled six new draft guidances outlining its latest thinking on the rapidly evolving field of gene therapy — and Commissioner Scott Gottlieb said the agency plans to work with product sponsors to help make the development and approval of innovative gene therapies more efficient.

The draft guidances deal with chemistry, manufacturing and control information for investigational new drugs, hemophilia, rare diseases, retinal disorders, long-term follow-up, and testing of retroviral vector-based therapies.

The CMC guidance outlines the necessary components of a gene therapy IND, including labeling and environmental analysis, noting that sponsors must provide a list of all materials used in manufacturing and a description of the quality and control of the materials.