The U.S. Food and Drug Administration approved PTC Therapeutics’ Sephience (sepiapterin) as an oral treatment for hyperphenylalaninemia in patients with phenylketonuria (PKU) aged one month and older who respond to sepiapterin. The broad label covers both pediatric and adult populations, offering a new option beyond strict dietary management for the rare metabolic disorder. The decision was supported by data from the Phase 3 APHENITY study and its extension, which showed sustained reductions in blood phenylalanine levels. Sephience is already authorized in the European Union, and review processes are under way in Japan and Brazil. Analysts expect the therapy to be a meaningful revenue driver; Jefferies forecasts peak annual sales of roughly $741 million by 2030. PTC shares rose about 8% after the announcement, and Citi raised its price target to $50 while maintaining a neutral rating.
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$PTCT +8.2% [FDA approved SEPHIENCE, an oral drug, for PKU treatment in children & adults 1 month+. It reduces phenylalanine, offering a new treatment option for sepiapterin-responsive patients. Jefferies expects peak annual sales of $741M from SEPHIENCE by 2030.]
Streetinsider.com PTC Therapeutics $PTCT Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
stock setter $PTCT - PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) - https://t.co/iFHBhv8X5a
