Tag Archive for: orphan drug designation

Poolbeg Pharma plc – Poolbeg granted FDA Orphan Drug Designation for POLB 001

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27 May 2025 – Poolbeg Pharma (AIM: POLB, ‘Poolbeg’ or the ‘Company’), a clinical-stage biopharmaceutical company focussed on the development of innovative medicines to address unmet medical needs, announces that the US Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) to POLB 001 as an oral preventative therapy for T-cell engager bispecific antibody-induced Cytokine Release Syndrome (“CRS”). Read more…

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Kaerus Bioscience’s lead candidate KER-0193 granted Orphan Drug Designation and Rare Pediatric Drug Designation by U.S. FDA for treatment of Fragile X syndrome (FXS)

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FDA’s two designations demonstrate KER-0193’s potential as a treatment for FXS Follows the successful completion of Phase 1 clinical trial Fragile X syndrome is the most common cause of inherited autism London, UK, 14 May 2025 – Kaerus Bioscience (“Kaerus” or “the Company”), a clinical stage biopharmaceutical company created by Medicxi for the development of […]

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U.S. FDA Grants Orphan Drug Designation to Ariceum Therapeutics’ Proprietary Radiopharmaceutical Cancer Therapy

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225Ac-satoreotide is a first-in-class Actinium-labelled somatostatin receptor 2 antagonist targeting extensive-stage Small Cell Lung Cancer or Merkel Cell Carcinoma Orphan Drug Designation follows outstanding preclinical data and FDA IND clearance Phase I/II human trials set to commence in Q1 2025 as a ‘theranostic’ targeted radionuclide treatment Berlin, Germany, 6 February 2025 – Ariceum Therapeutics (Ariceum), a […]

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Heidelberg Pharma granted orphan drug designation by FDA for its proprietary ATAC candidate HDP-101

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Ladenburg, Germany, 27 March 2024 – Heidelberg Pharma AG (FSE: HPHA), a clinical stage biotech company developing innovative Antibody Drug Conjugates (ADCs), is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the treatment of multiple myeloma to its lead candidate HDP-101. Heidelberg Pharma is investigating the […]

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US FDA grants Orphan Drug Designation for iOnctura’s first-in-class autotaxin cancer therapy

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Autotaxin inhibitor, IOA-289, recently received the proposed INN cambritaxestat Cambritaxestat is in the Phase I AION-02 trial in combination with chemotherapy in metastatic pancreatic cancer It is the first autotaxin inhibitor to be investigated in cancer patients Amsterdam, The Netherlands and Geneva, Switzerland, 7 March 2024 – iOnctura, a pioneering, clinical-stage biotechnology company developing transformative cancer […]

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