Clinical Trial RobustnessEfficacy signals come from small, open-label studies, which raises uncertainty about whether the results will be replicated in larger, controlled trials.
Development And Regulatory Risk For New IndicationsExpanding into indications such as Prader-Willi syndrome requires additional safety and efficacy evidence, adding development and regulatory risk to the program.
Safety Data SufficiencyLimited patient exposure leaves questions about long-term tolerability and the need for more comprehensive safety data before broad physician adoption.