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PTC's DMD drug fails to meet main goal in key late-stage study
October 15, 2015 / 8:08 PM / 2 years ago

PTC's DMD drug fails to meet main goal in key late-stage study

Oct 15 (Reuters) - PTC Therapeutics Inc’s experimental muscle disorder drug failed to meet the main goal in a key late-stage study, but the company said data from all trials on the drug supported its U.S. marketing application.

The drug failed to show statistically significant benefit over a placebo in a keenly watched trial on patients with Duchenne muscular dystrophy (DMD), for which there is no therapy approved by the U.S. Food and Drug Administration.

However, in a sub-group of less advanced patients, the drug Translarna’s effect was deemed clinically meaningful.

“Without a doubt these results confirm - from the totality of data - Translarna’s ability to slow disease progression, and we are actively planning for a U.S. launch in 2016,” Chief Financial Officer Shane Kovacs told Reuters on Thursday.

DMD is a progressive degenerative disorder that hampers muscle movement and affects one in 3,600 newborn boys, most of whom die by age 30. (Reporting by Natalie Grover in Bengaluru; Editing by Savio D‘Souza)

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