Tag Archive for: rare disease

BIO-Europe C-Suite Survey: Biopharma dealmaking set to accelerate, but investor caution still caps ambition

75% of biopharma leaders expect partnering and M&A appetite to increase over the next 12 months, none expect a decline 69% expect access to capital to improve, yet 79% identify investor risk aversion as the biggest constraint on biotech investment 52% say US policy uncertainty has made Europe more attractive to build and partner in, […]

NEZGLYAL® (leriglitazone) receives a positive CHMP opinion for the treatment of cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease

Recommendation based on NEXUS data; Minoryx and Neuraxpharm expect European Commission approval by the end of this quarter Barcelona, Spain and Düsseldorf, Germany – 24 July, 2026 – Minoryx Therapeutics and Neuraxpharm Group, announce that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional […]

4SC Files Marketing Authorisation Application with EMA for Resminostat (Kinselby)

Planegg-Martinsried, Germany, 1 March 2024 – 4SC AG (4SC, FSE Prime Standard: VSC), a biotech company improving the lives of patients suffering with advanced-stage cutaneous T-cell lymphoma (CTCL), announced today that it has filed its Marketing Authorisation Application (MAA) for resminostat (Kinselby) with the European Medicines Agency (EMA) and that its MAA has been accepted by […]

Poolbeg Pharma plc – Independent Research Confirms >$10bn Market Opportunity for POLB 001 as a Preventative Therapy for Cancer Immunotherapy-Induced Cytokine Release Syndrome (CRS)

Former Amryt team’s arrival supports increasing focus upon Rare and Orphan Diseases 12 February 2024 – Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, ‘Poolbeg’ or the ‘Company’), a biopharmaceutical company focused on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, today announces that independent research[i] [ii] conducted on behalf of Poolbeg confirms a >$10 billion market opportunity […]

Calliditas Therapeutics announces full FDA approval of TARPEYO®, the only FDA-approved treatment for IgA nephropathy to significantly reduce the loss of kidney function

Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”), today announced that the U.S. Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. TARPEYO was first approved in December 2021 under accelerated […]

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