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

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DUBLIN, Ireland, Dec. 27, 2017 — Horizon Pharma plc (NASDAQ:HZNP) today announced the U.S. Food and Drug Administration (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 pediatric 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.“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, head of kidney diseases and the Isaac A. Abt MD professor of kidney diseases, The Ann and Robert H. Lurie Children’s Hospital of Chicago, as well as professor of pediatrics at Northwestern University Feinberg School of Medicine.  “As a physician who conducts research and also cares for kids with nephropathic cystinosis, I’m particularly excited about the outcome of the clinical study and the subsequent PROCYSBI labeling 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 (WBC) cystine levels from poor controlled to well controlled at 12 and 18 month measurements.  WBC levels are the biomarker for disease control.  Additionally, they experienced measured improvements in growth milestones including weight and height.  The most common adverse reactions (>10%) 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.  This study was required by the FDA as a post-marketing commitment after PROCYSBI was approved. “The expanded PROCYSBI indication and revised prescribing information provide important guidance for physicians caring for people living with nephropathic cystinosis, particularly those treating very young children,” said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc.  “We are grateful for the families who enrolled their children in the clinical study that resulted in this update to the prescribing information – their participation led to the availability of new data that may improve the treatment of children living with nephropathic cystinosis.”About Cystinosis
Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.  If untreated, elevated cystine accumulation leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.  It is estimated that only about 2,000 people worldwide are currently diagnosed with nephropathic cystinosis.  Nephropathic or “classic infantile” cystinosis – the most common and most severe form of the disease – is typically diagnosed in infancy and requires lifelong cystine depleting therapy.1
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