FDA Officials Announce New Pathway for Bespoke Gene Therapies

Scientists created a customized gene editing therapy for a baby, which corrected the genetic variants, researchers said.

The U.S. Food and Drug Administration in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

Top Food and Drug Administration officials on Nov. 12 said there’s a new pathway for custom gene editing therapies for people with serious diseases.

The FDA will consider clearing bespoke therapies if a manufacturer shows success with several patients, Dr. Marty Makary, the FDA’s commissioner, and Dr. Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, said in an article published by the New England Journal of Medicine.

After the FDA grants authorization under the “plausible mechanism pathway,” companies will have to gather real-world evidence confirming that a therapy is effective and showing there were no unexpected risks.

Companies must also study how the therapies impact the growth and developmental milestones of children.

“Although the FDA will prioritize rare diseases, particularly those that are fatal or associated with severe disability in childhood, a plausible mechanism pathway will be also available for common diseases, particularly diseases for which there are no proven alternative treatments or in which there is considerable unmet need after use of available therapy,” Makary and Prasad wrote.

“For instance, a single disease with 150 different genetic mutations with the same functional implication may require 150 different therapies, and the plausible mechanism pathway would be ideally suited to such therapies. An appropriately designed study with a small sample size can support licensure of a product for which pharmacologic effect is aligned with biologic plausibility and congruent with observed clinical outcomes. That philosophy, in essence, embodies the plausible mechanism pathway.”

The officials highlighted the case of an infant who was diagnosed with a genetic deficiency disease that has a 50 percent mortality rate. Scientists created a customized gene editing therapy for the baby, which corrected the genetic variants that caused the disease, researchers said in a paper published by the same journal.

While critics have said that current FDA pathways are sufficient for bespoke therapies, patients, parents, researchers, and others have told the FDA they are too onerous and demanding, the officials said. They said they share that view.

“Nearly 30 years after the sequencing of the human genome, bespoke therapies are close to reality. The FDA will work as a partner and guide in ushering these therapies to market, and our regulatory strategies will evolve to match the pace of scientific advances,” the officials said.

The Alliance for Regenerative Medicine, which promotes cell and genetic therapies, welcomed the development.

“This modernization of the FDA’s regulatory approach will help U.S. patients benefit from groundbreaking scientific advances,” the group said on X. “We encourage the FDA to drive this vision with formal guidance that will enable its implementation with consistency, clarity, & appropriate oversight.”