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orphan drug designation

  • Biotech & Pharma Business,Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas

    AB2 Bio receives $30 million as Nippon Shinyaku exercises US option for rare disease drug

    AB2 Bio will receive $30 million from Nippon Shinyaku after [...]

    July 30, 2026
  • Advanced Therapies,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    Chiesi and Arbor win EU orphan status for PH1 gene editing therapy

    The European Commission has granted orphan drug designation to Chiesi [...]

    June 28, 2026
  • Biotech & Pharma Business,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    Mabion revives MabionCD20 through rare disease collaboration with Oddifact

    Mabion has signed a letter of intent with French technology [...]

    June 16, 2026
  • Clinical & Regulatory,Drug Delivery & Formulation,Pharmaceuticals and therapeutics,Therapeutic Areas

    FDA grants orphan drug designation to Tris Pharma’s TRN-257 for idiopathic hypersomnia

    FDA has granted orphan drug designation to Tris Pharma’s investigational [...]

    June 11, 2026
  • Clinical & Regulatory,Therapeutic Areas

    Rare Disease Day 2026: Cereno Scientific advances disease-modifying therapies in PAH

    As Rare Disease Day 2026 shines a spotlight on patients [...]

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Cereno Scientific completes expanded access program milestone for CS1 in pulmonary arterial hypertension

    Cereno Scientific has completed the last patient’s last visit in [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

    EMA grants orphan drug designation to Kedrion treatment for congenital aceruloplasminemia

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

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

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

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

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    SOM Biotech secures EMA orphan designation opinion for Huntington’s drug SOM3355

    Barcelona-based biotech advances Huntington’s programme as EMA backs orphan designation, [...]

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Cereno taps global CRO for next big step in pulmonary hypertension drug trial

    Selection of CRO marks key milestone in advancing CS1 as [...]

    May 5, 2026
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