The FDA is not a fan of PTC Therapeutics’ (PTCT) Duchenne muscular dystrophy drug.
The biotech company, based in South Plainfield, N.J., is trying to convince the FDA to approve the drug, known as Translarna, to treat a type of Duchenne muscular dystrophy caused by a so-called nonsense mutation. But on Tuesday, Translarna was rather robustly savaged in an FDA clinical review posted to the agency’s web site.
“Ultimately, no positive results from any prospectively planned analyses that are persuasive have been provided with this application,” the FDA review states.
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