Published: Tue, September 20, 2016
Economy | By Melissa Porter

FDA Oks 1st muscular dystrophy drug; awaits proof it works

FDA Oks 1st muscular dystrophy drug; awaits proof it works

The U.S. Food and Drug Administration on Monday granted preliminary approval to the first drug for a rare form of muscular dystrophy.

The approval comes almost five months after the Food and Drug Administration and a panel of outside advisers panned the drug, saying there was little evidence that it helped.

Approval to market eteplirsen was given to pharmaceutical company Sarepta Therapeutics.

Company shares have received an average consensus rating of Hold for the current week Sarepta Therapeutics (NASDAQ:SRPT): stock was range-bound between the intraday low of $27.9901 and the intraday high of $29.93 after having opened at $29.4 on Fridays session. Tim Lugo, an analyst with William Blair, estimated the drug will generate global peak annual sales of close to $2 billion. The company reported ($1.35) earnings per share for the quarter, missing the consensus estimate of ($1.19) by $0.16. Without that protein, muscles weaken so that children are unable to walk and must use wheelchairs by the time theyre teens.

Eteplirsen was initially set to come before the FDA's Peripheral and Central Nervous System Drugs advisory committee (adcom) in January, where it faced a hostile reception from FDA reviewers. Before that panel meeting, FDA staffers had indicated they were "prepared to be flexible with respect to a devastating illness with no treatment options".

The agency usually follows the recommendation of its advisers, whose split vote against approval was met with angry outbursts at the conclusion of a long and contentious meeting in April.

This is the second time in just over a year that the FDA has bowed to patient pressure to approve a drug despite scant scientific evidence showing it worked.

Several companies have pulled out of the space after the FDA made clear there was no path forward for approval for their drugs. Dr. Ellis Unger, a senior physician in the drug division, objected to her decision and filed a protest that reached Dr. Califf, the commissioner.

Nonetheless, Califf chose to "defer to Dr. Woodcock's judgment and authority to make the decision". Sarepta is still required to conduct a clinical trail to confirm the drug's benefits, and the FDA could withdraw its approval of the drug if the trial fails to show clinical benefits.

"The approval of Exondys 51 reflects FDA's ability to apply flexibility to address challenges we often see with rare, life-threatening diseases - while remaining within our statutory framework", Woodcock said in that letter.

The FDA said it wouldn't make any of its staff available for interviews.

Dr. John Lee, chief medical officer of the rare disease biotech company PhaseBio, said the FDA's decision on Sarepta is a boon to the whole field.

As a reward for bringing a drug for a rare pediatric disease to market, Sarepta also receives an FDA voucher to speed up the review of another drug.

"If this drug can be approved under those conditions, is there any drug that FDA wont' approve?" said Diana Zuckerman, president of the National Center for Health Research, a nonprofit research organization. "This is the outcome MDA dreamed of 25 years ago when it was the first to invest in the breakthrough research that led to development of eteplirsen".

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