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NEWS

PTC Therapeutics Receives the Health Canada Approval for Sephience to Treat Phenylketonuria

Shots: Health Canada has approved PTC's Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in pts (≥1mos.) with sepiapterin-responsive PKU Approval was based on APHENITY study results & long-term extension data showing durable effects & favorable safety in PKU pts Sephience is a natural precursor…
December 11, 2025

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BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

Shots: The US FDA has accepted sBLA & granted priority review to Palynziq for the treatment of adolescents (12-17yrs.) with phenylketonuria (PDUFA: Feb 28, 2026) sBLA was supported by the P-III (PEGASUS) trial comparing Palynziq to diet alone in 55 adolescents with phenylketonuria, which showed reduced blood phenylalanine (Phe); results were presented at ICIEM’25 Palynziq…
October 31, 2025

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NEWS

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…
July 29, 2025

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PTC Therapeutics
NEWS

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…
April 25, 2025

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