Accelerated Approval For Gene Therapies - US Health Regulator Looks To Improve Process

Vandana Singh

March 21, 2023 at 4:16 PM·2 min read

The FDA seeks to optimize the development of gene therapies to help get accelerated approval of therapies.
The US health regulator is looking to encourage using biomarkers, such as pulse and blood pressure, which are characteristics of the body that can be measured.
The FDA will support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help clinch "accelerated approval," Reuters reported citing the agency's official Peter Marks.
FDA grants accelerated approval to drugs for rare/serious diseases.
The agency still needs confirmatory trials to verify clinical benefits. Approval of a drug may be withdrawn or the labeled indication of the drug changed if trials fail to verify clinical benefit or do not demonstrate sufficient clinical benefit.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 side effects over the long term and safety during administration.
Adding that post-marketing tools such as safety monitoring and the possible use of extra clinical trials will be key.
Sarepta Therapeutics Inc (NASDAQ: SRPT) is seeking accelerated approval for Duchenne gene therapy, for which the FDA recently announced to hold an adcomm meeting less than a month after saying it would not do so.
Photo via Wikimedia Commons

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