Ann: Acadia submits Rett Syndrome New Drug Application to the FDA, page-20

HT, thank you for that link. For me it's reassuring that Neuren /Acadia have proven both safety and efficacy of Trofinetide in regrads to the take-away para in the article;

This study found that FDA refusal to file NDAs and efficacy supplements for review is rare. When applications are refused for review, however, it is for numerous substantive reasons associated primarily with missing information needed to evaluate product quality, safety, and efficacy. Notwithstanding that missing scientific and technical matters accounted for 84.5% of refusal reasons, we found that the public disclosure of these RTF reasons is minimal. Safety and effectiveness assessment lies at the heart of the FDA’s drug review, yet 44.7% of RTF letters were issued owing to missing efficacy data, and 26.2% of RTF letters cited missing both safety and efficacy data. This finding shows that RTF letters address foundational issues in drug review.15