Tag Archive for: rare diseases

Poolbeg Pharma plc – Exclusive Option Agreement to Acquire Orphan Drug Candidate for Behçet’s Disease

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Novel therapeutic with Fast Track and Orphan Designation 30 April 2024 – Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, ‘Poolbeg’ or the ‘Company’), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces that it has entered into an exclusive 12-month option agreement with Silk Road Therapeutics Inc,  for […]

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Poolbeg Pharma – Results for the year ended 31 December 2023

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Significant pipeline advancements Strategic expansion into cancer immunotherapy-induced CRS unlocking a market opportunity exceeding US$10Bn Key senior management hires 30 April 2024 – Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, ‘Poolbeg’ or the ‘Company’), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces its audited results for the […]

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Calliditas announces an additional seven year orphan drug exclusivity period for TARPEYO®

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Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the FDA has granted an orphan drug exclusivity period of seven years for TARPEYO®, expiring in December 2030 based on when the company obtained full approval with a new indication for this drug product. Following full approval in December 2023, TARPEYO® (budesonide) is indicated […]

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Vivet Therapeutics receives EUR 4.9 million to advance development of a gene therapy

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Vivet Therapeutics receives EUR 4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis  Funding from French Government as part of the France Health Innovation Plan 2030 operated by Bpifrance  Paris, France, February 1, 2024 – Vivet Therapeutics (“Vivet”), a clinical stage biotech company developing novel and long-lasting gene therapies for […]

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Calliditas Therapeutics announces full results from the NefIgArd Phase 3 trial published in The Lancet

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Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced publication in The Lancet of the full data from the Phase 3 NefIgArd Study with Nefecon® (TARPEYO® (budesonide) delayed release capsules/Kinpeygo®) in adults with Primary IgA nephropathy (IgAN). The Phase 3 trial met the primary endpoint, estimated glomerular filtration rate (eGFR), with Nefecon demonstrating significant kidney protective effect over […]

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