Horizon Pharma receives FDA approval for Procysbi Label Expansion

Horizon Pharma Plc. (NASDAQ: HZNP) announced today the U.S. Food and Drug Administration has approved an expansion for its Procysbi (cysteamine bitartrate) delayed- release capsules to include children of the age one year and older suffering from nephropathic cystinosis.

According to the National Institute of Health, nephropathic cystinosis begins in infancy, causing poor growth and a particular type of kidney damage (renal Fanconi syndrome) in which certain molecules that should be reabsorbed into the bloodstream are instead eliminated in the urine. Untreated children will experience complete kidney failure by about the age of 10.

Procysbi, which has already been approved for adults and children aged two and older, is a delayed-release form of cysteamine bitartrate that reduces the toxic concentration of cystine in the cells. With the updated indication, Procysbi prescriptions include revised guidelines for physicians administering the drug to young children.

“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 and head of kidney diseases and the Isaac A. Abt MD professor of kidney diseases“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.”

Horizon’s shares have underperformed the industry year to date. The stock has declined 7.2% against the industry’s growth of 3.5%.

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