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POSTPONED FOR INCLEMENT WEATHER: Pediatric Advisory Committee and Endocrinologic and Metabolic Drugs Advisory Committee

The PEDAC and EMDAC will meet to discuss the major objectives of a phase 3 drug development program indicated for the treatment of children with achondroplasia (ACH). The following elements of a phase 3 program should be considered for discussion: evidence required to establish dose-response, study design, e.g., placebo control, study duration, intended population, e.g., infants and toddlers and/or older children and adolescents, and endpoints that have a clinically meaningful impact on the patient’s functional or psychological well-being.