Exclusive: Dr. Ron Farkas, Eteplirsen's Harshest Critic, Has Left The FDA

The "miracle drug" that could save the lives and well-being for hundreds of people may have just received some great news.

Dr. Ron Farkas has left the U.S. Food & Drug Administration, people familiar with the matter told Benzinga. Why would his departure raise hope for the "miracle drug" -- eteplirsen -- produced by Sarepta Therapeutics Inc SRPT?

Update: At 10:16 a.m. ET, a FDA spokesperson confirmed to Benzinga that Farkas has left the administration.

"A 13-member FDA advisory committee voted against approving eteplirsen, a treatment for Duchenne muscular dystrophy by Sarepta Therapeutics, concluding that there wasn't sufficient evidence the drug was effective," Wall Street Journal opined in April. Farkas was the drug's most vocal critic, although there were many other FDA members who agreed there was little efficacy for eteplirsen.

Farkas had issues "with the randomized controlled trials (two phase 2 studies and one phase 3 study) and open-label extension studies conducted by the sponsor."

Shares of Sarepta spiked more than 12 percent in Tuesday's after-hours session, when Jenn McNary Tweeted at 5:13 p.m. ET, "Seems like Ron Farkas is no longer at the @US_FDA."

Shortly after, Benzinga confirmed Farkas was no longer listed in the FDA's Silver Springs directory. A source told Benzinga that Farkas' last day was Friday, September 9.

McNary has two children who suffer from Duchenne Muscular Dystrophy (DMD) and has been one of eteplirsen's strongest advocates. Farkas' departure would "suggest approval" for eteplirsen.

The Street's Adam Feuerstein said there was another side to the rumor -- Farkas joined Parexel PRXL. A senior director at Parexel told Benzinga he wasn't aware of Ron Farkas.

Sarepta's stock closed at $25.60, down 7.7 percent on the day.

Update: On Monday, September 19, the FDA cleared eteplirsen for accelerated approval. The stock spiked 80 percent on the news.

Posted In: Dr. Ron FarkasDuchenneDuchenne Muscular DystrophyeteplirsenJenn McNaryRon FarkasBiotechNewsHealth CareFDATop StoriesMarket-Moving ExclusivesExclusivesHotGeneral