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- 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 Approval of a drug may be withdrawn or the labeled indication of the drug changed if trials fail to verify clinical benefit or do not demonstrate sufficient clinical benefit.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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