The U.S. Food and Drug Administration (FDA) has released several new gene therapy guidance documents to provide “regulatory clarity” for developers of the many investigational therapies designed to treat any number of chronic conditions, including those for hemophilia. The FDA emphasized their firm commitment to supporting innovation in the interest of patients while reiterating the importance of establishing a safe path through clinical development and potential approval. The need for comprehensive guidance is critical considering the field’s many rapid advancements, as evidenced by the sheer volume of investigational new drug (IND) applications coming to the agency – the FDA reports receiving 900 IND applications for ongoing gene therapy clinical trials in many therapeutic areas. 

“The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators,” said FDA Commissioner Stephen M. Hahn, MD “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.”

“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,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “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.”

The guidance documents are open to the public and can be readily accessed at no cost via the FDA website. Follow the links below to view these documents.

Human Gene Therapy for Rare Diseases – Guidance for Industry

Human Gene Therapy for Hemophilia – Guidance for Industry

Long Term Follow-Up After Administration of Human Gene Therapy Products – Guidance for Industry

Additional guidance documents can also be found on the FDA website

Source: FDA news release dated January 28, 2020