27 December 2017 - Horizon Pharma today announced the U.S. FDA has approved an expansion to the indication for Procysbi (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis. 

With this update to the indication, the Procysbi prescribing information now includes revised guidance for physicians administering the medicine to paediatric patients, including new clinical evidence and dosing information for very young children. Procysbi was previously FDA-approved for adults and children as young as two years of age.

The Procysbi labeling was updated based on a long-term, prospective, open-label study that enrolled 17 people living with nephropathic cystinosis, including 15 children between the ages of 1 and 5 years old, who had not previously been treated with cysteamine therapy. Children enrolled in the study experienced lowering of white blood cell (WBC) cystine levels from poor controlled to well controlled at 12 and 18 month measurements. WBC levels are the biomarker for disease control.

Read Horizon Pharma press release