Results from a study on Procysbi delayed-release capsules in treatment-naïve children younger than 6 years of age with nephropathic cystinosis.- Horizon Pharma.
Horizon Pharma plc announced results from a new open-label study evaluating the effects of Procysbi (cysteamine bitartrate) delayed-release capsules in treatment-naïve children younger than 6 years of age living with nephropathic cystinosis. These data were presented at the American Society of Nephrology (ASN) Kidney Week 2017 Annual Meeting in New Orleans on Saturday, 4 November. In the United States, Procysbi is a cystine depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients two years of age and older.
In the study, children enrolled achieved lowered and prolonged maintenance of white blood cell (WBC) cystine levels – the biomarker for disease control – over the course of one year of therapy. Additionally, they experienced measured improvements in height, weight and body surface area (BSA).
Summary of Study Results: This long-term (minimum of 12 months), prospective, controlled, open-label study enrolled 17 people living with nephropathic cystinosis, including 15 children between the ages of 1 and 4.5 (mean age: 2.2 years).The primary objective was to determine the long-term safety and effectiveness of Procysbi on WBC cystine levels among patients naïve to prior cysteamine treatment. The percentage of patients who reached WBC cystine levels of less than 1.0 nmol ½ cystine/mg increased over the treatment period. While the study was not designed to evaluate changes in height, weight, and body surface area, the results support further investigation of the effect of Procysbi on these measures. Mean height, weight and BSA increased over the treatment period.
The most common adverse reactions (greater than 5%) in patients treated in clinical trials reflected in the FDA approved product labeling were vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash and headache.
Comment: This study was required by the FDA as a post-marketing commitment after Procysbi was approved. The results of this study are not included in the FDA approved product labeling for Procysbi. Procysbi is not approved for use in children less than two years of age.
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