FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

Delivery Might Be Key to Gene Therapy’s Orphan Status, CBER Official Says

May 13, 2019
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The FDA will consider granting orphan status for different gene therapies—even ones in the same class as a previously designated orphan treatment—based on how the therapy is delivered, a high-ranking CBER official said.

The agency is willing to look at “the totality of the circumstances around the product,” said Julie Tierney, CBER’s senior advisor for strategic planning and legislation, in a panel discussion on orphan drug designations at the Food and Drug Law Institute’s annual meeting in Washington, D.C. She said the FDA wants to be as flexible as possible and doesn’t view orphan decisions as a constant “head-to-head” competition.

“There can be a lot of differences based on how you’re delivering the genetic modification,” Tierney told FDAnews. “There can be different clinical profiles, different safety issues, different effectiveness, so how you’re delivering the therapy can be just as important as the gene you’re going for.”

Last year, the FDA received 209 INDs for gene or other advanced therapies, about double the number for the previous year, Tierney said.

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