The US FDA approved Procysbi (cysteamine bitartrate) delayed-release capsules on 30 April and is indicated for adults and children with cystinosis aged six years and more.
Initial prescription claims have been approved by payors and are now being made available at Accredo Specialty Pharmacy, according to the company.
Comprising 95% of cases of cystinosis, Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs of the body.