PTC Therapeutics Announces New Sephience™ (sepiapterin) Data Reinforcing Broad Patient Benefits Including Sustained Metabolic Control and Diet Liberalization as well as Consistent Favorable Safety Profile
PTC Therapeutics announced that 17 abstracts featuring Sephience (sepiapterin) data will be presented at the SSIEM Annual Symposium, demonstrating sustained metabolic control and significant diet liberalization for PKU patients. New analyses from the AMPLIPHY study indicate a 100% greater reduction in blood Phe for participants switching from sapropterin to Sephience, with meaningful reductions observed in high-baseline patients within 14 days.
Key figures
- Baseline phe threshold
- ≥900 µmol/L
- Pku patient population
- 58000
- Response timeframe days
- 14
- Normalization blood phe level
- <120 µmol/L
- Blood phe reduction vs sapropterin
- 100%
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