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Home>UPDATE: Investors want PTC Therapeutics to be the next Sarepta

UPDATE: Investors want PTC Therapeutics to be the next Sarepta

UPDATE: Investors want PTC Therapeutics to be the next Sarepta

10/02/2017

By Emma Court

Company shares surged 14% though most members of an FDA advisory committee voted 'maybe'

After a Food and Drug Administration advisory committee voted "maybe" on its Duchenne muscular dystrophy drug, PTC Therapeutics Inc. shares have surged -- gaining 10% after hours on Thursday and 13.7% in extremely heavy trade Friday.

FDA decisions about drug approval don't have to follow the advisory committee's recommendation. But they typically do -- with a major, and recent exception: biotech Sarepta Therapeutics Inc.'s (SRPT) DMD drug Exondys 51 (http://www.marketwatch.com/story/sareptas-controversial-duchenne-muscular-dystrophy-drug-was-contested-right-up-to-approval-2016-09-19).

Ever since Exondys 51 was approved over an advisory committee's negative vote and internal FDA concerns, there have been questions about whether the controversial decision would set a precedent (http://www.marketwatch.com/story/sareptas-positive-trial-results-for-new-dmd-treatment-could-reopen-rare-disease-drug-controversy-2017-09-06).

Probably not this time, Wall Street said. Based on Thursday's meeting, "we think the most likely outcome is a [Complete Response Letter] with need for a new trial," said RBC Capital Markets analyst Matthew Eckler, referring to the FDA's way of declining to approve a drug.

Read: PTC Therapeutics gains after FDA panel votes against drug approval (http://www.marketwatch.com/story/ptc-therapeutics-gains-after-fda-panel-votes-against-drug-approval-2017-09-28)

DMD is a rare genetic disease characterized by progressive muscle weakness. It primarily affects boys early in their childhood, and typically kills patients before age 30. Sarepta was the first U.S.-approved medication for DMD; PTC Therapeutics (PTCT) now owns Emflaza, another DMD drug dogged by controversy (http://www.marketwatch.com/story/why-ptc-therapeutics-may-have-to-lower-the-price-on-a-89000-a-year-drug-it-just-bought-2017-03-16).

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