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BioWorld - Friday, April 3, 2026

Orphan drug

Home » Topics » Regulatory » Orphan drug
  • Wood approved stamp red
    March 19, 2026
    By Karen Carey

    GSK’s IBAT inhibitor wins US FDA nod for cholestatic pruritus

    With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus.
  • Molecule illustration
    March 17, 2026
    By Marian (YoonJee) Chu

    Otsuka’s repinatrabit shows phase II efficacy in PKU adolescents

    Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
  • Molecule illustration
    March 13, 2026
    By Marian (YoonJee) Chu

    Otsuka’s repinatrabit shows phase II efficacy in PKU adolescents

    Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
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