FDA Advisers Say No To Duchenne Muscular Dystrophy Drug

The United States Food and Drug Administration (FDA) advisory panel voted against the approval of eteplirsen, a drug for Duchenne muscular dystrophy. The result came in the midst of petitions from patients, parents and doctors who badly sought for the drug's approval. They said that eteplirsen was allowing boys who had the disease to walk for an extended period of time beyond what was expected.

Ryan's dad speaking. No side effects. "Eteplirsen has given us a reason to hope." Teachers, friends, notice he is doing better. $SRPT

— Matthew Herper (@matthewherper) April 25, 2016

How Eteplirsen Works

Eteplirsen, manufactured by Sarepta Therapeutics, would have been the first drug to combat Duchenne muscular dystrophy. Eteplirsen employs a genetic technique called exon skipping, the New York Times said.

In exon skipping, a disease is cured or amended by correcting a defective gene. Sarepta has been talking with the FDA about eteplirsen since November 2013, Muscular Dystrophy News Today reported. This was also the period when the FDA had ruled that it was premature to have accelerated approval for eteplirsen.

How The Voting Went Down

Voting 7 to 3, with three abstentions, members voted that drug manufacturer Sarepta Therapeutics failed to meet FDA standards for well controlled studies essential for approval, the New York Times reported. Meanwhile, a closer vote of 7 to 6 were cast in terms of giving eteplirsen accelerated approval.

Sarepta Therapeutics' clinical study, which it used as basis for approval for the drug called eteplirsen, only had 12 patients, per the New York Times. The study also had no ample placebo control. Sarepta issued a statement about the decision, Yahoo reported.

BREAKING: $SRPT shares plummet 51% in pre-market after FDA panel says Sarepta's Duchenne drug not proven effective https://t.co/Ezs7VfNsfr — CNBC Now (@CNBCnow) April 26, 2016

"We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA," said Dr. Edward Kaye, Sarepta's interim CEO and chief medical officer. "Today more than ever, we remain committed to our mission of bringing a treatment to the Duchenne community."

The final decision on eteplirsen's approval still rests with the F.D.A. The F.D.A. will decide whether or not it will approve the drug on May 26.

Duchenne Muscular Dystrophy

According to the Muscular Dystrophy Association (MDA), Duchenne muscular dystrophy is "a genetic disorder characterized by progressive muscle degeneration and weakness." This is just one type of muscular dystrophy. There are nine types in all.

A lack of the protein dystrophin is what causes Duchenne muscular dystrophy. Dystrophin aids in keeping muscle cells free from any damage.