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Sephience

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Representative image illustrating the global expansion of phenylketonuria treatment, reflecting Japan’s approval of Sephience by PTC Therapeutics and the growing international reach of rare disease therapies.

Pharma & Biotech

PTC Therapeutics receives Japanese approval for Sephience in full-spectrum PKU

by Venkatesh B
December 22, 2025
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Japan approves Sephience for PKU across all ages. Find out what this means for PTC Therapeutics' global rare disease expansion strategy.

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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