PTC sees high­er reg­u­la­to­ry risk in fight to keep its Duchenne drug on the Eu­ro­pean mar­ket

PTC Ther­a­peu­tics $PTCT hasn’t yet lost the right to mar­ket its failed Duchenne mus­cu­lar dy­s­tro­phy drug Translar­na in Eu­rope. But the biotech’s ex­ec team is telling in­vestors that they have wan­ing hopes of con­tin­u­ing to make it avail­able as reg­u­la­tors pon­der whether they’ll re­new the au­tho­riza­tion.

In a state­ment out Wednes­day the com­pa­ny not­ed:

Fol­low­ing the com­pa­ny’s CHMP meet­ing in Oc­to­ber, the com­mit­tee re­quest­ed ad­di­tion­al in­for­ma­tion re­gard­ing the risk-ben­e­fit pro­file of Translar­na, its ef­fi­ca­cy and the de­sign of a po­ten­tial tri­al that would pro­vide com­pre­hen­sive clin­i­cal da­ta. While we have pro­vid­ed in­for­ma­tion in re­sponse to the CHMP’s re­quests and ex­pect to con­tin­ue to en­gage in fur­ther in­ter­ac­tions, re­cent di­a­logue has in­tro­duced a high­er de­gree of un­cer­tain­ty as to the out­come. 

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