UPDATE 2-EU panel recommends against PTC Therapeutics drug

Fri Jan 24, 2014 11:28am EST

(Adds detail from CHMP statement, analyst comments; updates share movement)

By Vrinda Manocha

Jan 24 (Reuters) - PTC Therapeutics Inc said its drug to treat a rare muscular disorder failed to win support for a conditional approval from a panel of the European Medicines Agency, sending its shares down as much as 17 percent in morning trading.

The European Committee for Medicinal Products for Human Use said a study failed to show that patients taking the drug, ataluren, could walk a greater distance in six minutes than patients on placebo. (link.reuters.com/vew36v)

It also said the drug showed limited evidence of having beneficial effects on the daily activities of patients with a form of Duchenne muscular dystrophy (DMD), a genetic disorder that causes muscle degeneration and eventual death in boys.

Analysts said conditional approval for the drug had been considered unlikely, and remained positive on its potential for eventual approval by the EMA.

A conditional approval is granted to treatments that address unmet medical needs. The drugmaker is still testing the drug in a late-stage trial.

The EMA was concerned that conditional approval would make it difficult for the company to finish the trial, Wedbush analyst Christopher Marai said.

PTC said it plans to request the committee for a re-examination, but analysts said they expect the company would need data from its late-stage trial of the drug before it is approved.

Marai said the drug could be approved by late 2015 or early 2016 and would target about 1,500 patients in the European Union. "The drug could generate peak sales of $350 million by 2019 in the European Union," he said.

PTC joined a list of companies struggling with setbacks to their drugs to treat DMD.

Prosensa Holding NV's partner GlaxoSmithKline Plc handed back the rights to its DMD drug less than two weeks ago after the failure of a key trial.

Sarepta Therapeutics Inc lost almost two-thirds of its market value in November after the U.S. FDA said the design and goals of its trial might not be sufficient to get approval for its muscular disorder drug.

About 15 percent of DMD cases are caused by nonsense mutations that leads to a premature end to the translation of a gene into a protein, according to the U.S. National Institutes of Health.

Ataluren is designed to interact with a part of the cell known as a ribosome, enabling it to manufacture a protein called dystrophin, the absence of which causes muscle weakening in patients with DMD.

The company's shares were trading down 4 percent at $24.20 in morning trading on the Nasdaq. (Reporting by Vrinda Manocha in Bangalore; Editing by Don Sebastian)

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