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Fri, Jul 11, 2025

Orphan drug

Home » Topics » Regulatory » Orphan drug
  • Multiple myeloma illustration
    July 4, 2025

    Brightpath’s BP-2202 designated orphan drug for multiple myeloma

    Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.
  • Dna and cancer cells
    July 1, 2025

    Hemispherian’s GLIX-1 awarded US orphan drug designation for malignant glioma

    GLIX-1 is a first-in-class small-molecule therapeutic targeting DNA repair vulnerabilities in cancer cells.
  • 3d bispecific antibodies
    June 19, 2025

    Diagonal Therapeutics’ DIAG-723 designated orphan drug for hereditary hemorrhagic telangiectasia

    Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.
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