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Welcome to Discover Pharma! Today is : April 25, 2026

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Orphan drugs

  • Clinical Development,Drug Development,FDA,Genetic Diseases,Orphan drugs,Pharmaceuticals and therapeutics,Rare Diseases

    BridgeBio submits FDA NDA for BBP-418 in limb-girdle muscular dystrophy type 2I/R9

    BridgeBio Pharma has submitted a New Drug Application (NDA) to [...]

    March 31, 2026
  • Clinical Trials,Oncology,Orphan drugs,Pharmaceuticals and therapeutics,Research & Development

    Oncoinvent to present 24-month Phase 1 ovarian cancer data for Radspherin at ESGO 2026

    Oncoinvent will present final 24-month follow-up data from its Phase [...]

    February 18, 2026
  • Orphan drugs,Rare Diseases,Regulatory Affairs

    EMA grants orphan drug designation to Kedrion treatment for congenital aceruloplasminemia

    Kedrion has received orphan drug designation from the European Medicines [...]

    November 27, 2025
  • Cell & Gene Therapy,Oncology,Orphan drugs,Regulatory Affairs

    Minovia Therapeutics gains FDA orphan drug designation for MNV-201 in myelodysplastic syndrome

    Minovia Therapeutics has received orphan drug designation from the US [...]

    October 17, 2025

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