Tag Archive for: rare disease

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 […]

Vicore Pharma: Interim report July-September 2023

Stockholm, November 2, 2023 – Vicore Pharma Holding AB (STO: VICO) (“Vicore”), unlocking the potential of a new class of drug molecules – angiotensin II type 2 receptor agonists (ATRAGs), publishes the interim report for the third quarter 2023. Read more…

Raising awareness of Rare Disease

Each year, Rare Disease Day aims to raise awareness of rare diseases and encourage positive change to patients’ lives. A disease is considered rare when it affects fewer than 1 in 2,000 people and there are 300 million people with rare diseases across the world.1 On Rare Disease Day 2023, we want to celebrate the […]