- U.K. health-cost regulator recommends Translarna in England
- Drug application had been rejected by FDA in February
PTC Therapeutics Inc. soared the most since May 2014 after the U.K.’s health-cost regulator recommended its drug for a deadly muscle disease.
The U.K.’s National Institute for Health and Care Excellence, known as NICE, recommended PTC’s drug Translarna for some patients with Duchenne muscular dystrophy in England, according to a statement Friday. PTC jumped 49 percent to $9.26 at 12:07 p.m. in New York.
The recommendation is a boon for PTC, whose Translarna application to the U.S. Food and Drug Administration was rejected in February, sending the shares down 62 percent on a single day.
Whether patients will get the drug will depend on NICE’s final draft guidance, which the regulator expects next month, PTC said. Financial terms are still being finalized, according to the statement.
PTC had fallen 81 percent this year as of the close on Thursday, dragged down by the FDA rejection. PTC has also faced difficulties in Europe, where Translarna is conditionally approved in Europe. The company has said it’s considering pulling the drug from Germany’s pharmacy ordering system because it didn’t see the outcome of pricing arbitration with the country’s health system as acceptable. The drugmaker said last month it would fire about 18 percent of its workers.
The FDA has been skeptical about how effective recent experimental treatments for DMD are. Another biotechnology company working on a drug for DMD, Sarepta Therapeutics Inc., last month said it would cut about 17 percent of its workforce.
DMD is a rare disease that occurs in about one of 3,500 boys born worldwide, according to the U.S. National Institutes of Health. It causes muscle degeneration at an early age, warping bones and swelling the heart. Few patients live past their 30s.