(Reuters) – The U.S. Food and Drug Administration needs to start using accelerated approval for advancing gene therapies for rare diseases, STAT News reported on Monday, citing agency official Peter Marks.
Accelerated approval would be particularly important for ultra-rare diseases, for which there are too few patients to run placebo-controlled studies, the report said, quoting Marks, who heads the agency’s Center for Biologics Evaluation and Research.
“When you’re making a gene therapy for 10, 20 people a year, the concept that you’re going to do a randomized clinical trial falls apart pretty quickly,” Marks said, according to the report.
The FDA did not immediately respond to a Reuters request for comment.
The U.S. health regulator grants the so-called “accelerated approval” for drugs and therapies mainly for 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.
Sarepta Therapeutics Inc said late last week that the FDA planned to hold a panel meeting to review the company’s 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.
One of the measures that Marks proposed to get gene therapies for rare disease patients was a new regulatory standard, STAT News reported.
(Reporting by Raghav Mahobe in Bengaluru; Editing by Shilpi Majumdar)
