Tag Archive for: ODD

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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European Commission Grants Orphan Drug Designation for Vivet Therapeutics Gene Therapy Product for the Treatment of Cerebrotendinous Xanthomatosis (CTX)

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Pre-clinical data of VTX-806 in mice demonstrated normalization of metabolic parameters in blood and brain, and of upregulated compensatory enzymes EUR 4.9 million funding provided by French Government to develop VTX-806 as an effective treatment option to stabilize or reverse symptoms in CTX patients Paris, France, September 5, 2024 – Vivet Therapeutics (“Vivet”), a clinical […]

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4SC provides Q3 highlights and financial forecast

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Planegg-Martinsried, Germany, 19 October 2023 – 4SC AG (4SC or “the Company”) (4SC, FSE Prime Standard: VSC), a biotech company improving the lives of patients suffering with advanced-stage cutaneous T-cell lymphoma (CTCL), today provided a business update for the three months ended 30 September 2023, as well as its current outlook. The full report is available […]

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4SC receives Orphan Drug Designation (ODD) for resminostat (Kinselby) in CTCL in the European Union

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The European Medicines Agency (EMA) has granted Orphan Drug Designation for resminostat for treatment of cutaneous T-cell lymphoma (CTCL) ODD benefits include 10 years’ market exclusivity in the European Union The Marketing Authorisation Application (MAA) for Kinselby (resminostat) in the EU remains on track for filing in Q1 2024 Planegg-Martinsried, Germany, 18 October 2023 – 4SC […]

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