SEPHIENCE™ (sepiapterin)

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PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
Prnewswire· 2025-07-28 20:19
Core Viewpoint - PTC Therapeutics, Inc. has received FDA approval for SEPHIENCE™ (sepiapterin) to treat phenylketonuria (PKU), marking a significant advancement for patients with this rare metabolic disorder [1][2][3] Group 1: FDA Approval and Product Details - SEPHIENCE is approved for treating hyperphenylalaninemia (HPA) in patients aged 1 month and older with sepiapterin-responsive PKU [1][6] - The approval is based on positive results from the Phase 3 APHENITY trial, demonstrating significant efficacy and safety [2][3] - SEPHIENCE acts as a natural precursor of the enzymatic co-factor BH4, effectively reducing blood phenylalanine levels [5] Group 2: Market Potential and Community Impact - The broad labeling of SEPHIENCE reflects its potential to address the unmet needs of PKU patients, with expectations to become the future standard of care [2] - The approval is seen as a milestone for the PKU community, providing renewed hope for improved quality of life for affected individuals [3] - SEPHIENCE has also received marketing authorization from the European Commission, with ongoing reviews in countries like Japan and Brazil [3] Group 3: Company Strategy and Future Plans - PTC Therapeutics is prepared to launch SEPHIENCE in the U.S. swiftly, leveraging its expertise in rare disease therapies [2] - The company plans to hold a conference call to discuss the approval and its implications for the market [4][9] - PTC aims to optimize value for patients through its diversified pipeline of transformative medicines for rare disorders [14]