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Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies

Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market. Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that may be available to sponsors of these therapies.

Today, we’re unveiling a complementary framework for the development, review and approval of gene therapies. In the past 12 months, we’ve seen three separate gene therapy products approved by the FDA. This reflects the rapid advancements in this field.


Statement From FDA Commissioner Scott Gottlieb, M.D. On Agency’s Efforts To Advance Development Of Gene Therapies