(Reuters) -Shares of Sarepta Therapeutics Inc slumped as much as 21% on Friday over uncertainty on approval of its gene therapy for Duchenne muscular dystrophy after the U.S. health regulator reversed its decision on a panel meeting.
The U.S. Food and Drug Administration planned to hold a meeting of its independent experts to review the gene therapy, the company said late on Thursday, less than a month after saying it would not do so.
“This change adds drama to the equation,” said Baird analyst Brian Skorney. He added the FDA’s inconsistency may be a signal of more internal debate than previously anticipated.
The company does not expect a delay as the FDA is working to schedule a meeting ahead of the May 29 action date, Sarepta said in a conference call on Thursday.
Sarepta is developing SRP-9001 as the first gene therapy for the treatment of Duchenne muscular dystrophy, an inherited disorder of progressive muscular weakness typically seen in boys. The company already has three marketed drugs targeting Duchenne patients with certain genetic mutations.
Gene therapies such as BioMarin’s bleeding disorder therapy have faced setbacks and tough scrutiny in the past, but the FDA approved multiple such products in 2022. Gene therapies typically carry high prices as they are often one-time treatments. Recently approved products from bluebird bio and Australia’s CSL were priced at $2.8 million and $3.5 million, respectively.
Roche holds the right to launch and commercialize SRP-9001 outside the U.S.
Massachusetts-based Sarepta said in February the FDA did not plan a meeting, which helped shares post their biggest one-day percentage gain in over three years.
The company’s shares were trading at $118.5, 3% below the price before the February announcement.
(Reporting by Aditya Samal; Editing by Krishna Chandra Eluri and Sriraj Kalluvila)
