FDA’s Marks Calls for Providing Accelerated Approval Path for Gene Therapies
March 21, 2023
Rare Daily Staff
Peter Marks, director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research, said the agency will support the use of biomarkers to provide for the use of accelerated approval for gene therapies.
The comments came during the annual meeting of the Muscular Dystrophy Association and were reported in STAT.
The U.S. Food and Drug Administration’s Accelerated Approval pathway allows for the use of surrogate endpoints to make therapies more quickly available for unmet medical needs. About 82 percent of the drugs approved under the designation have been for orphan indications. But controversy around its use to win approval for Biogen’s Alzheimer’s disease drug Aduhelm last year set lawmakers off on an effort to reform how the pathway is used and to place new requirements on drugmakers to complete confirmatory studies in a timely manner.
In a statement provided to Reuters, Marks noted that for some gene therapies, there may be a need to “accept some level of uncertainty” at the time of approval, but that post-marketing tools such as safety monitoring and the possible use of extra clinical trials are going to be key.
The comments come days after the agency reversed itself and said it would hold an advisory committee meeting for Sarepta Therapeutics’ experimental gene therapy for Duchenne muscular dystrophy, which the agency is expected to act on by May 29.
Photo: Peter Marks, director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research
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