Sarepta Soars After FDA Staffer’s Exit Lifts Approval Hopesby and
Ron Farkas, reviewer critical of Sarepta drug, departed
Sarepta still awaits FDA decision on muscle disease treatment
Sarepta Therapeutics Inc. rose the most since June after a U.S. Food and Drug Administration reviewer who was critical of the drugmaker’s proposed treatment left the agency, potentially brightening prospects for approval of the medicine.
Ron Farkas is no longer at the FDA, according to an e-mail on Wednesday from Sandy Walsh, a spokeswoman. Farkas led a review that criticized studies of Sarepta’s drug, called eteplirsen, that was developed to treat a lethal muscle disease. He started working at Parexel International Corp., a consulting firm, earlier this month, according to spokeswoman Cristi Barnett.
Sarepta began rising late Tuesday after multiple posts on Twitter and a Benzinga report said that Farkas had left the FDA. The shares rose 26 percent to $32.17 at 12:16 p.m. in New York, the biggest intraday increase since June 7.
Sarepta is developing eteplirsen to treat Duchenne muscular dystrophy, a degenerative disease that affects young boys. An FDA panel voted in April that there wasn’t enough evidence to show whether the drug is effective. Patients and their families have pleaded on behalf of the drug, saying that it has helped prolong their ability to walk and stand, and the agency has delayed its decision on approval.
In documents prepared for the April panel, Farkas wrote that results from one of Sarepta’s trials were “essentially uniformly negative, without trends supportive of efficacy” and questioned how much eteplirsen helped to increase levels of the muscle protein dystrophin, which is missing in people with DMD. The drug is designed to treat about 13 percent of patients with the disease.
Sarepta is trying to be first to market with a treatment for DMD, a genetic disease in which patients’ ability to walk worsens quickly and eventually leads to breathing difficulties. Patients often die by age 25, usually from lung disorders, according to the National Institutes of Health.
The FDA has already rejected two other DMD drug candidates, from Biomarin Pharmaceutical Inc. and PTC Therapeutics Inc. Biomarin ended its program, while PTC is continuing to seek U.S. approval after the FDA said its application wasn’t sufficiently complete to permit a substantive review. Shares of PTC gained 9.1 percent to $9.14.
Sarepta is seeking accelerated approval, which allows the drug to go to market based on trial results that show an effect on patients that’s reasonably likely to predict benefit. If the FDA clears eteplirsen for sale, Sarepta would have to do more studies on the drug to prove its benefit and gain regular approval.