Vertex Pharmaceuticals announced positive Day 90 data from the first patient in the company’s Phase I/II study of VX-880 in type 1 diabetes (T1D). The therapy is a stem cell-derived, fully differentiated pancreatic islet cell replacement therapy.
The U.S. Food and Drug Administration approved Xeris Pharmaceuticals Inc.’s glucagon pen Gvoke, which aims to treat severely low sugar levels in diabetes patients.
In people at high risk for type 1 diabetes, 14 days of therapy with the experimental drug teplizumab delayed development of the disease by a year or more.
EU panel approves drug for rare genetic disease
Blood, Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (EMA), Familial Chylomicronemia Syndrome (FCS), Fat Accumulation, FDA, FDA/Regulatory, Inflammation, Pancreas, Platelet Count, Rare Genetic Diseases, Recommended For ApprovalAn advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.