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Yet another company developing a drug for Duchenne muscular dystrophy suffered a setback as PTC Therapeutics disclosed yesterday that the Food and Drug Administration rejected its application for its experimental treatment. The move is also the latest disappointment for the small, but highly vocal patient advocacy community that is pushing the agency very hard to approve a drug for the rare disease.

The FDA sent the drug maker a so-called Refusal to File letter, which means its application to win marketing approval was insufficient to permit a substantive agency review. The disclosure triggered a big sell-off in PTC stock and, more broadly, prompted a renewed round of skepticism over whether the FDA will endorse any drugs to treat Duchenne muscular dystrophy.


In recent weeks, the FDA declined to approve a drug from BioMarin Pharmaceutical and released documents in advance of an advisory committee meeting scheduled for last month in which agency medical reviewers suggested a Sarepta Therapeutics treatment was unlikely to be approved. Sarepta, however, quickly provided additional information, prompting the agency to delay the meeting until May.

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  • This administration’s track record for giving the go ahead for new drugs is not very good. Must not fit their ‘agenda’. If this drug had been for one of their select groups – .001% of the population who wanted to use a bathroom that they ‘identify with ‘ in their minds, but not with their bodies though THAT goes through because they are ‘progressive thinkers’.

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