Lipid disorders Archives

Orphazyme’s Rare Lipid Disorder Drug Gets FDA Priority Review

Accepted NDA, Clinical Data, Clinical Trials, Fast Track Designation, FDA, FDA/Regulatory, Heat-Shock Proteins (HSPs), Lipid disorders, Niemann-Pick C Disease (NPC), Primary Endpoints, Priority Review Status, R&D, Rare Genetic Diseases, Secondary Endpoints

The U.S. Food and Drug Administration accepted Copenhagen, Denmark-based Orphazyme’s New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C.

September 16, 2020/by Andrew Humphreys

Waylivra Approved In EU For Treating FCS

ApoC-III, Approvals, European Commission, Familial Chylomicronemia Syndrome (FCS), FDA/Regulatory, Genetic Diseases, Lipid disorders, Lipoprotein Lipase (LPL), Triglycerides, Ultra Rare Diseases

Akcea Therapeutics Inc. and Ionis Pharmaceuticals Inc. announced that Waylivra received conditional marketing authorization from the European Commission as the only therapy for FCS.

May 7, 2019/by GlobeNewswire