Erasmus MC
🇳🇱Netherlands
European Commission Approves Emcitate (Tiratricol) as First Treatment for MCT8 Deficiency
• The European Commission has granted marketing authorization for Emcitate (tiratricol), making it the first approved treatment for monocarboxylate transporter 8 (MCT8) deficiency in the EU.
• The approval is based on data from Triac Trial I, the Erasmus University Medical Center Cohort Study, and preliminary results from Triac Trial II, demonstrating significant reductions in serum T3 concentrations.
• Emcitate is indicated for treating peripheral thyrotoxicosis in MCT8 deficiency patients from birth, addressing symptoms like increased heart rate and muscle weakness.
• Egetis Therapeutics anticipates initiating pricing and reimbursement discussions in Europe, with the first launch expected in the second quarter of 2025.
Active Biotech's Tasquinimod Trial for Myelofibrosis Receives European Approval
• Active Biotech's Phase I/II trial (HOVON 172 MF) of tasquinimod for myelofibrosis patients resistant to or intolerant of JAK2 inhibitors has been approved by the EMA and ethics committees.
• The open-label, multicenter study will assess tasquinimod's safety and efficacy as a monotherapy, with a primary endpoint of a ≥35% reduction in spleen volume after six cycles.
• The trial, conducted within the HOVON network in the Netherlands and Germany and financed by Oncode Institute, is expected to begin enrollment shortly.
• Tasquinimod, an immunomodulator, has shown disease-modifying potential in preclinical myelofibrosis models and is also in trials for multiple myeloma.
ASN Kidney Week: Promising Clinical Trial Results Highlight Advances in Kidney Disease Treatment
• A trial showed targeted correction of hyponatremia in hospitalized patients did not improve mortality and rehospitalization rates.
• Pegcetacoplan significantly reduced proteinuria in patients with glomerulopathy in the VALIANT trial, showing favorable safety.
• Semaglutide reduced major kidney outcomes in type 2 diabetes patients with chronic kidney disease, irrespective of CKD severity.
• Acellular tissue-engineered vessels showed higher success rates in maintaining vascular access for hemodialysis, especially in high-risk patients.
ASN Kidney Week 2024: Clinical Trials Highlight Advances in Kidney Disease Treatment
• A trial showed targeted correction of hyponatremia in hospitalized patients did not improve mortality and re-hospitalization rates.
• Pegcetacoplan significantly reduced proteinuria in patients with complement 3 glomerulopathy or primary immune complex-mediated membranoproliferative glomerulonephritis.
• Semaglutide reduced major kidney outcomes, cardiovascular events, and death risks in type 2 diabetes patients with chronic kidney disease, irrespective of CKD severity.
• Dapagliflozin slowed kidney function decline and reduced kidney/cardiovascular outcomes in advanced chronic kidney disease patients.
MesoPher Immunotherapy Shows Promise in Resected Pancreatic Cancer
• A phase 2 trial of MesoPher, a dendritic cell-based immunotherapy, demonstrated a 64% 2-year recurrence-free survival rate in patients with resected pancreatic cancer.
• The study also reported an 83% 2-year overall survival rate, suggesting a clinically meaningful immune response against the tumor.
• MesoPher has received orphan drug designation from both the FDA and EMA for pancreatic cancer, potentially expediting its development and regulatory review.
• A randomized phase 2/3 trial is being planned to further evaluate the efficacy of MesoPher in resected pancreatic cancer.
Long-term efficacy and safety of iptacopan in PNH with anaemia
The article discusses the long-term efficacy and safety of iptacopan, a treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) with anaemia, as presented by Prof. Antonio Risitano from the University of Naples, Italy.
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