FDA Releases Six New Draft Guidances on Gene Therapy

July 12, 2018

On 7/11/2018 FDA Commissioner, Scott Gottlieb, announced the release of six new draft guidance documents related to gene therapy development. In the announcement, Gottlieb acknowledged the accelerating pace of development and highlighted the FDA's role in both aiding development and ensuring safety.

Gottlieb also discussed the FDA's current and evolving approach to regulating the development of new gene therapies.  “We look forward to working with the academic and research communities to make safe and effective products a reality for more patients. But we know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time. In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval,” he said.

The six new draft guidance documents

Human Gene Therapy for Hemophilia

Human Gene Therapy for Retinal Disorders

Human Gene Therapy for Rare Diseases

Chemistry, Manufacturing, and 2 Control (CMC) Information for 3 Human Gene Therapy Investigational 4 New Drug Applications (INDs)

Testing of Retroviral Vector-Based 2 Human Gene Therapy Products for 3 Replication Competent Retrovirus 4 During Product Manufacture and 5 Patient Follow-up

Long Term Follow-Up After Administration of Human Gene Therapy Products

Gottlieb also mentioned that the FDA would, “make full use of our expedited programs such as breakthrough therapy designation and regenerative medicine advanced therapy designation whenever possible.”






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