Horizon Pharma plc announces FDA approval to expand the indication for Procysbi (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis.
The U.S. Food and Drug Administration (FDA) has approved an expansion to the indication for Horizon Pharma plc’s Procysbi (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis.
With the update to the indication, the Procysbi prescribing information now includes revised guidance for physicians administering the medicine to pediatric patients, including new clinical evidence and dosing information for very young children. It was previously FDA-approved for adults and children as young as two years of age.
“Data included in the updated label provide further evidence around the unique role of Procysbi in helping physicians manage young children during one of the most crucial periods for growth,” said Craig B. Langman, M.D., lead investigator for the study that led to the label update.
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 cystine levels from poor controlled to well controlled at 12 and 18 month measurements. Additionally, they experienced measured improvements in growth milestones including weight and height.
The most common adverse reactions (>10 percent) in patients treated in clinical trials reflected in the FDA approved product labeling were vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, and headache. The study was required by the FDA as a post-marketing commitment after Procysbi was approved.
(Source: Horizon Pharma plc)
Filed Under: Drug Discovery