FDA approves Procysbi (Raptor Pharmaceutical) for Nephropathic Cystinosis

The FDA has approved Procysbi (cysteamine bitartrate) delayed release capsules, from Raptor Pharmaceutical, for the treatment of Nephropathic Cystinosis in adults and children 6 years and older. The approval was based on a New Drug Application (NDA) comprising data from six clinical trials, including a multi-center randomized, active-controlled Phase III trial of 43 patients with Nephropathic Cystinosis and extension data from that trial. The most commonly reported adverse reactions were vomiting, abdominal pain, discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash.

Clinical studies are ongoing in children less than six years of age to evaluate for the first time the safety and efficacy of the drug. Over ninety percent of the Cystinosis population is six years or older.