FDA Releases Guidance on Gene Therapy Manufacturing, Clinical Development

PRICENTRIC BRIEF:

• The Food and Drug Administration has approved 4 gene therapy products thus far, and anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area
• To support the continued development of gene therapies, the FDA is announcing the release of six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
• The six final guidances 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on gene therapy products to address specific disease areas to help advance the field of gene therapy, while the draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease

THE DETAILS

WASHINGTON, D.C., The United States – The Food and Drug Administration has approved 4 gene therapy products thus far, and anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area.

To support the continued development of gene therapies, the FDA is announcing the release of six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The six final guidances 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 and help advance the field of gene therapy while providing recommendations to ensure that these innovative products meet the FDA’s standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality, and the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs (e.g. questions about the durability of response).Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations, which 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, focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

Through this, 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).

FDA Commissioner Stephen M. Hahn, M.D, said, “The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.”

Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said, “As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.”

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