PTC Therapeutics announced on October 1, 2024, that the U.S. Food and Drug Administration (FDA) accepted for filing its New Drug Application (NDA) for sepiapterin. This application seeks approval for the treatment of pediatric and adult patients living with phenylketonuria (PKU), covering all age groups and disease subtypes.
The NDA is supported by highly statistically significant and clinically meaningful results from the Phase 3 APHENITY trial. This trial demonstrated a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in the classical PKU subgroup.
Furthermore, 84% of subjects achieved Phe control below 360 µmol/L, with 22% showing normalization of Phe levels. Data from the APHENITY open-label extension study and a Phe tolerance sub-study also indicated that approximately 60% of subjects could increase protein intake above the recommended daily allowance while maintaining Phe control.
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