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.

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

A U.S. government-backed panel says there is not enough evidence to say all children should have blood tests to check for high levels of fat-like substances known as lipids.