PTC Therapeutics, Inc. · lettre de réponse complète (NDA 220049)

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ADMINISTRATION ¢ YN U.S. FOOD & DRUG NDA 220049 COMPLETE RESPONSE PTC Therapeutics, Inc. Attention: Aparna Geddam, MS Director, Global Regulatory Strategy 500 Warren Corporate Center Drive Warren, NJ 07059 Dear Aparna Geddam: Please refer to your new drug application (NDA) oe or vatiquinone oral capsules, oe We have completed our review of this application, as amended, and have determined that we cannot approve this application in its present form. We have described our reasons for this action below and, where possible, our recommendations to address these issues. CLINICAL/BIOSTATISTICS 1. After a detailed review of your application, we have concluded that the data do not demonstrate substantial evidence of effectiveness (SEE) for vatiquinone in the treatment of Friedreich’s Ataxia (FA). You proposed to meet the evidentiary standard based on a single adequate and well-controlled study with confirmatory evidence. The single controlled trial supporting this application, PTC742-NEU- 003-FA (MOVE-FA), is not capable of serving as a single adequate and well- controlled study, with or without confirmatory evidence (CE), to provide SEE. The deficiencies in the data are outlined below: e MOVE-FA failed to demonstrate a statistically significant difference between vatiquinone and placebo for the prespecified primary endpoint of change from baseline to week 72 in the modified Friedreich’s Ataxia Rating Scale (mFARS). Change from baseline in mFARs is an outcome measure that i

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