Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

Key Points

• Sepiapterin significantly lowers blood Phe levels compared to sapropterin, highlighting its efficacy.
• No new safety signals were reported during the trial, ensuring patient safety.
• This active-controlled trial compares two therapies in phenylketonuria, a genetic disorder affecting amino acids.
• Findings suggest sepiapterin may be a better treatment option, warranting further clinical exploration.