US FDA to take steps to help gene therapies get accelerated approval

[March 21, 2023]  (Reuters) -The U.S. Food and Drug Administration is moving to optimize the development of gene therapies, including encouraging the use of biomarkers to help get accelerated approval of therapies for serious conditions, an agency official said.

Biomarkers, such as pulse and blood pressure, are characteristics of the body that you can measure. The FDA will support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help clinch "accelerated approval", the agency's Peter Marks said on Monday.

The U.S. health regulator grants the so-called accelerated approval mainly for drugs and therapies targeted at rare diseases or small patient populations that have had no effective treatments available to them. Companies are still required to conduct studies to confirm the anticipated clinical benefit.

"The FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases," said Marks, who heads the FDA's Center for Biologics Evaluation and Research, in a statement.

For certain gene therapies, there may be a need to "accept some level of uncertainty" at the time of approval around questions like side-effects over long-term and safety during administration, the statement said, adding that post-marketing tools such as safety monitoring and the possible use of extra clinical trials are going to be key.

The comments from the FDA come days after Sarepta Therapeutics Inc said that the agency planned to hold a panel meeting to review its gene therapy for Duchenne muscular dystrophy, less than a month after saying it would not do so.

The company is seeking approval for its gene therapy under the FDA's accelerated pathway.

(Reporting by Raghav Mahobe in Bengaluru; Editing by Shilpi Majumdar and Devika Syamnath)

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