Tailor-made gene editing: FDA moves to ease approval of individualized therapies
The U.S. Food and Drug Administration proposes allowing many variants of a gene-editing therapy to be approved in a single application. Rare disease advocates welcomed the draft.
Symbolic image · Photo: Ernesto del Aguila III, NHGRI / Wikimedia Commons, Public domain (cropped and resized)
The U.S. Food and Drug Administration on Tuesday published draft guidance on individualized gene-editing therapies. It targets patients with rare mutations whose treatment must be tailored to their specific genetic change. Until now, each such product variant would have needed its own approval application, an approach that Andrew Byrnes, director of the FDA's Division of Gene Therapy 1, called "infeasible" and "not necessary."
At the core of the draft is a so-called individualized feature space. It defines the limits within which the gene-editing components, such as the guide RNA, may vary across all versions of a product. If a new variant falls within those limits and clinical or laboratory data predict adequate safety and activity, it would be treated as a manufacturing change rather than a new product. New variants could also be added after approval.
Rare disease advocates welcomed the move. "It's sort of putting a flag in the ground and saying, 'This is important,'" said Paul Scribner of the PKD Foundation, which supports people with polycystic kidney disease. The document is a draft and may still change.