PTC Therapeutics has managed to slash through the regulatory jungle to find a path forward for its Friedreich ataxia medication, vatiquinone, after a pair of failures last year left the biotech cutting staff and reprioritizing its pipeline.
PTC executives briefed investors on the results of a meeting with the FDA where they pled their case for using a subscale called upright stability to show efficacy using data already collected in a Phase III trial. While vatiquinone failed to move the needle on the modified Friedreich Ataxia Rating Scale (mFARS) - the therapy had little impact on lower and upper limbs - PTC saw a glimmer of efficacy in the data, pointing to the upright stability subscales. Click here to learn more.
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