Achondroplasia Trial Design: US FDA Panel Moves Patient-Centered Development To The Forefront
Advisory committee’s recommendations on secondary endpoints for pivotal studies reflects patient and caregiver comments during the open public hearing about the disease’s clinical complications, including ear infections and sleep apnea, and challenges performing activities of daily living.
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Very early launch numbers look good in France and Germany, but US launch will be more challenging due to a fragmented patient base, the company said. Launch price is a flat $899 per vial.
US FDA advisory committee had encouraged a two-year endpoint for achondroplasia treatments, but BioMarin attributes success to having ‘partnered closely’ with the agency. Confirmatory trial still being discussed.
Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials
The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.