FDA Promotes Gene Therapy with New Guidance Documents

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The agency has published seven guidance documents directed at the development and manufacture of gene therapies.

As part of the agency’s efforts to advance the development of gene therapies, FDA announced on Jan. 28, 2020 the publication of seven guidance documents regarding the development and manufacture of gene therapies. These documents are being issued to help advance the field of gene therapy while ensuring that these products meet safety and effectiveness standards, according to FDA.

Six of the documents are final guidance and provide recommendations for gene therapies to address specific disease areas. The final guidance are as follows:


The seventh document is a draft guidance on interpreting sameness of gene therapies under the orphan drug regulations. It describes how the agency will evaluate gene therapies and how it will consider the principal molecular structural features including transgenes and vectors. According to FDA, “With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations. The draft guidance has potential positive implications both for product developers and patients by providing insight into the agency’s most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition.”

“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, in a press release. “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.”

Source: FDA