Health Canada approves Horizon Pharma’s nephropathic cystinosis drug

Health Canada has issued a Notice of Compliance (NOC) for Horizon Pharma’s Procysbi (cysteamine delayed-release capsules) for the treatment of nephropathic cystinosis in adults and children 2 years of age and older. 

“For children diagnosed with nephropathic cystinosis, treatment with cystine-depleting therapy should be started as soon as possible to avoid the serious and potentially life-threatening impact on tissues and organs,” said Durhane Wong-Rieger, president and chief executive officer, Canadian Organization for Rare Disorders (CORD).  “Prior to Procysbi there was no approved therapy in Canadafor treating cystinosis.  We are especially pleased that Health Canada has granted very timely approval of Procysbi, the delayed-release form of the therapy, which will help assure patients avoid toxic build-up of cystine in the cells.”

The company said that Procysbi is the only cystine-depleting agent approved in Canada for the treatment of nephropathic cystinosis.  It is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells to limit or prevent the damage too much cystine can cause to cells, tissue and organs.

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.  Elevated cystine 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 cystinosis.  Nephropathic or “classic infantile” cystinosis is the most common and most severe form of the disease, and is typically diagnosed in infancy and requires lifelong therapy.