PTC Therapeutics' shares double on positive CHMP decision for DMD drug Translarna

Shares in PTC Therapeutics more than doubled Friday after the European Medicines Agency's Committee for Medicinal Products for Human Use reversed an earlier decision and backed conditional approval for the Duchenne muscular dystrophy (DMD) therapy Translarna (ataluren). PTC chief executive Stuart W. Peltz, remarked that the recommendation "is a major milestone for the DMD community," with the company noting that the drug is "the first treatment for the underlying cause" of the disease.

In January, the CHMP issued a negative opinion regarding conditional approval for Translarna, with PTC requesting a re-examination of the decision. The EMA noted that during the re-assessment, the CHMP determined that there was "some evidence of effectiveness" for Translarna and the way the medicine "works is plausible." The CHMP said the safety profile of Translarna "was not of concern," concluding that given the "seriousness" of DMD and the unmet medical need of patients with this condition, the benefits of PTC's drug outweigh its risks.

The CHMP's recommendation, which is for the treatment of nonsense mutation DMD in ambulatory patients aged five years and older, is based on data from a Phase IIb study including 174 patients. According to PTC, results showed that patients treated with Translarna walked on average 31.3 meters farther than patients given placebo, as measured by the change in six-minute walk distance (6MWD) from baseline to week 48. In addition, the trial demonstrated that patients receiving Translarna had a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD.

Peltz commented that "as previously disclosed, we expect to have all patients enrolled in our global Phase III ACT DMD by mid-2014." The executive noted that "the outcome of this trial is critical for achieving full approval in the EU as well as the US."

Other companies developing treatments for DMD include Sarepta Therapeutics, which announced plans last month to apply to the FDA by the end of 2014 for authorisation to market its exon-skipping drug candidate eteplirsen.

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