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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