Arimoclomol for NPC receives Rare Pediatric Disease Designation

Orphazyme A/S, a Danish biotech company (ORPHA:CO), with a late-stage, orphan-drug pipeline, today announced that arimoclomol has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease Type C.
As quoted in the press release:

NPC is a genetic disease affecting around 1 in 120,000 newborns. Although the time of symptoms onset is variable, NPC is most often diagnosed in childhood and adolescence. Progressive neurological pathology is the hallmark of NPC and is responsible for disability and premature death in most patients. Arimoclomol, an orally available small molecule, is currently being investigated in a clinical Phase II/III trial as a potential treatment for NPC.

Click here to read the full press release.