Unfazed by failure, PTC Therapeutics to appeal negative EMA decision on its disaster prone Duchenne drug
PTC Therapeutics’ bid to expand the approval of its Duchenne muscular dystrophy drug in Europe has been unsuccessful. The New Jersey-based drugmaker is not known for taking no for an answer and will appeal the decision, it said on Friday.
The drug, ataluren, has a checkered past, littered with failed trials.
In Europe, the drug — branded as Translarna — was conditionally approved by the EMA in 2014 to treat patients aged 2 and older with DMD who are able to walk, just ahead of the readout of a late-stage study, which showed the drug had failed.
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