Raptor rises on US Procysbi OK
This article was originally published in Scrip
Executive Summary
Raptor Pharmaceuticals gained the US FDA's OK on 30 April to market Procysbi (cysteamine bitartrate) as a therapy for adults and children as young as six years to manage nephropathic cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.