PTC Therapeutics (NASDAQ: PTCT) has received FDA approval for SEPHIENCE™ (sepiapterin), a treatment for phenylketonuria (PKU) in patients aged 1 month and older. The approval covers broad labeling for hyperphenylalaninemia (HPA) in sepiapterin-responsive PKU patients.
The approval is supported by significant efficacy and safety data from the Phase 3 APHENITY trial and its long-term extension study. SEPHIENCE has already received European Commission marketing authorization, with approval applications under review in Japan and Brazil.
The company aims to position SEPHIENCE as the future standard of care for PKU patients, with their experienced teams ready to launch the therapy in the United States.
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