Despite miss in biomarker study, PTC to discuss FDA approval pathway based on totality of data

PTC’s Translarna may have missed the endpoints in its Duchenne muscular dystrophy biomarker study, but the company believes clinical measures from the trial, data from its other studies and real-world evidence could support FDA approval.

PTC Therapeutics Inc. (NASDAQ:PTCT) reported Thursday after market close that Translarna ataluren failed to raise dystrophin protein levels from baseline in biopsies from 20 patients ages 2-7 in the intent-to-treat population of Study 045. On the primary endpoint, dystrophin levels measured via electrochemiluminescence (ECL) went up by 6.56% (p=0.24); on the secondary endpoint, immunohistochemistry (IHC) assays showed a 4.91% increase (p=0.11)...