On January 28, 2020, the U.S. Food and Drug Administration (FDA) issued six final guidances on gene therapy manufacturing and clinical development of products as well as a draft guidance for industry, “Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.”

From the FDA: “The six final guidances issued today provide the agency’s recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies. The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDA’s standards for safety and effectiveness.

“The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDA’s proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity. The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the draft guidance and our regulations, the agency’s determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).”

To read the full news release, visit FDA.gov.

To learn more about the new draft guidance, visit regulations.gov.