FDA Commissioner Scott Gottlieb, MD, announced the framework, which is intended to be complementary to the comprehensive policy framework for regenerative medicine that was announced last year.
Gene therapies have the potential to treat and cure even some of the most intractable of diseases, and the FDA recently released a new framework for the development, review, and approval of gene therapies.
FDA Commissioner Scott Gottlieb, MD, announced the framework, which is intended to be complementary to the comprehensive policy framework for regenerative medicine that was announced last year. Over the course of the past year, 3 different gene therapy products have been approved by the FDA, reflecting the advancements in the area.
“Today, we’re taking a step toward shaping this modern structure for the regulation of gene therapy,” Gottlieb said in a statement. “The agency is issuing a suite of  scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.”
The 3 new draft guidance documents are the following:
The FDA also made updates to 3 existing guidance documents that address manufacturing issues related to gene therapy:
When these updated guidance documents are finalized, they will replace previous versions issued by the FDA.
“Gene therapy represents one of the most promising opportunities for developing highly effective and even curative treatments for many vexing disorders,” Gottlieb concluded. “Some of these products are almost certainly going to change the contours of medical practice, and the destiny of patients with some debilitating diseases.”