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PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

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…

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PTC Therapeutics

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU) 

Shots:  The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein  Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…

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