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BioWorld - Wednesday, January 7, 2026

Orphan drug

Home » Topics » Regulatory » Orphan drug
  • Genetic mutation illustration
    Dec. 16, 2025

    Immusoft’s ISP-002 designated US orphan drug for MPS II

    Immusoft of CA Inc., a wholly owned subsidiary of Immusoft Corp., has announced that the FDA has granted orphan drug designation to ISP-002, the company’s investigational engineered B-cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
  • Europe pills syringe flag
    Dec. 15, 2025
    By Nuala Moran

    EU agrees to pharma legislation, but the industry isn’t happy

    The EU finally reached agreement on an update of the 20-year-old pharmaceutical legislation, more than five years after the EU Commission first put forward the case for reform and following two and a half years of negotiations on the new rules.
  • Dec. 2, 2025
    By Marian (YoonJee) Chu

    Vigencell’s cell therapy for lymphoma meets phase II endpoint

    Vigencell Inc. plans to seek conditional approval in South Korea for VT-EBV-N, an antigen-specific killer T-cell therapy for natural killer T-cell lymphoma, after gaining positive top-line data from a phase II study Nov. 25.
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