Shots:
- The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & Brazil
- Approval was based on APHENITY study results & long-term extension data showing durable effects and improved dietary flexibility
- Sephience is an oral synthetic sepiapterin with a dual mechanism that boosts phenylalanine hydroxylase (PAH) enzyme activity
Ref: PRNewswire | Image: PTC Therapeutics| Press Release
Related News:- PTC Therapeutics Receives the EC’s Approval for Sephience to Treat Phenylketonuria (PKU)
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