FDA announces new measures to boost gene therapy innovation
The US Food and Drug Administration (FDA) has announced new policies to advance the development of safe and effective cell and gene therapies.
The announcement was made in a press release issued yesterday, January 15.
FDA commissioner Scott Gottlieb said the agency would try to make “maximum use” of its Regenerative Medicine Advanced Therapy (RMAT) designation. The scheme is an expedited approval programme for cell therapies which treat serious or life-threatening conditions. The FDA will now look to expand the scheme to include more gene therapies as well.
Gottlieb noted that many gene therapies carry risks associated with the products’ durability and potential for rare instances of off-target effects. Robust postmarket tools, included in the RMAT designation, "can help achieve the goal of developing a larger data set to address these theoretical risks in a timely fashion”, Gottlieb said.
The FDA will also issue a set of guidance documents in the coming year intended to advance the development of new gene therapies. These include therapies designed to treat conditions such as rare blood disorders and neurodegenerative diseases.
Gottlieb also expressed his concern that “a number of individuals” in the sector were operating outside of regulatory compliance and creating significant safety concerns in the production of cell therapies. The FDA will announce additional enforcement measures this year, he said.
The plans to boost the innovation of gene therapies are just the latest strategic measures announced by the FDA. In December, Gottlieb announced a programme to advance the development of medical devices.
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