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US Health Secretary Kennedy looks to fast-tracking approvals for rare disease drugs
By Sneha S K and Sriparna Roy - 6/5/2025
By Sneha S K and Sriparna Roy
(Reuters) -Health and Human Services Secretary Robert F. Kennedy Jr. said on Thursday that the U.S. drugs regulator would look for ways to fast-track approval for rare disease treatments and remove obstacles to their path to market.
Kennedy made the comments at a U.S. Food and Drug Administration meeting to discuss cell and gene therapies, where panelists called for faster regulatory processes as they warned that other countries may overtake the U.S. in drug development.
"We are going to continue to figure out new ways of accelerating approvals for drugs and treatments that treat rare diseases, and we're going to make this country the hub of biotechnology innovation," Kennedy said.
Other members included industry executives, researchers and FDA staffers, among them Vinay Prasad, the FDA's top vaccine and biologics official.
The appointment of Prasad as the head of the FDA's Center for Biologics Evaluation and Research had stoked fears that he could raise the bar for companies to get approval for new drugs, including what are known as accelerated approvals for new potential treatments of serious conditions.
Prasad vowed at the meeting to rapidly make therapies available at the first sign or promise of biomedical success or action.
Shares of therapy developers Sarepta, Dyne Therapeutics and Lexeo Therapeutics were trading between 1% and 3% higher in afternoon trading. U.S.-listed shares of uniQure rose 8.19% to $16.18.
Panel members said that the slower regulatory process for rare disease treatments risks the United States' position as a leader in the biotechnology sector at a time when drug development in China is accelerating.
"The path to approval is seen as so arduous. If firms feel there is no credible way to get new products approved here, they will simply relocate trials overseas or abandon them," panel member Carl June from University of Pennsylvania.
"We cannot afford that exodus," said June.
(Reporting by Sneha S K and Sriparna Roy in Bengaluru; Editing by Alan Barona)