Health
PTC Therapeutics Announces Health Canada Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month old and older with sepiapterin-responsive PKU.
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Broad label includes all age groups and full range of PKU subtypes -","length":70,"tagName":"p","attribs":{}},{"type":"text","content":"TORONTO, Dec. 10, 2025 /CNW/ - PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month old and older with sepiapterin-responsive PKU.","length":379,"tagName":"p"},{"type":"image","alt":"PTC Therapeutics, Inc. Logo (PRNewsfoto/PTC Therapeutics, Inc.)","displaySize":"","headline":null,"caption":"PTC Therapeutics, Inc. Logo (PRNewsfoto/PTC Therapeutics, Inc.)","className":"","disableSlideshowImg":false,"size":{"original":{"width":400,"height":244,"url":"https://media.zenfs.com/en/cnwgroup.com/5c78d9ee565c8a63b9f458b2e6aeee63"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/5eVuXz172ddTcL9DxQFGFg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTcwNTtoPTQzMDtjZj13ZWJw/https://media.zenfs.com/en/cnwgroup.com/5c78d9ee565c8a63b9f458b2e6aeee63","width":400,"height":244}},"href":"https://mma.prnewswire.com/media/2582388/PTC_LOGO_Logo_Logo.html","hrefExternal":true,"rel":"nofollow"},{"type":"text","content":""We're pleased with the Canadian regulatory approval of Sephience and opportunity to provide access to this important therapy to children and adults living with PKU in Canada," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "There is a significant unmet medical need for new therapies that address a broad spectrum of PKU subtypes."","length":386,"tagName":"p"},{"type":"text","content":"The Health Canada approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.","length":208,"tagName":"p"},{"type":"text","content":""There continues to be a significant unmet need for new treatment options in PKU which can support improved dietary flexibility and management, a meaningful goal for those living with PKU in Canada and around the world," said Tanya Chute Nagy, Chief Executive Officer and President, Canadian PKU and Allied Disorders.","length":337,"tagName":"p"},{"type":"text","co...