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  • PKU drug Sephience receives orphan drug designation in KOR
  • by Eo, Yun-Ho | translator Alice Kang | Jul 7, 2025 06:10am
Gains attention as a drug for treating hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU)
Receives final approval in Europe¡¦ demonstrates efficacy in APHENITY trial

Sephience, a new drug for phenylketonuria (PKU), a rare metabolic disorder, has been designated as an orphan drug in Korea.

The Ministry of Food and Drug Safety recently announced so through a public notice. Specifically, the drug is indicated for the treatment of hyperphenylalaninaemia (HPA) in adult and pediatric patients with phenylketonuria (PKU).

Sephience (sepiapterin), which was developed by U.S. biopharmaceutical company PTC Therapeutics, recently received marketing authorization from the European Commission and is currently undergoing approval procedures with the U.S. FDA.

More specifically, the drug is an oral formulation of tetrahydrobiopterin, a critical enzyme cofactor involved in the met
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