The Office of Orphan Product Development at the Food and Drug Administration (FDA) granted orphan drug exclusivity for Raptor Pharmaceutical's Procysbi (cysteamine bitartrate) delayed-release capsules for the management of nephropathic cystinosis patients age six and older. The exclusivity period began on the date of FDA approval, April 30, 2013, and lasts seven years subject to certain exceptions.
"This decision was the outcome of a productive and cooperative process with the FDA. We believe the issuance of orphan drug exclusivity highlights an appreciation that Procysbi is an important new therapeutic option for nephropathic cystinosis patients," stated Thomas E. Daley, chief business officer of Raptor. "The exclusivity also adds incremental protection alongside our issued patents listed in the FDA's Orange Book with expirations in 2027."
Nephropathic cystinosis comprises 95% of cases of cystinosis, 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. Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life. There are an estimated 500 patients living in the US with cystinosis and 2,000 worldwide.
Cystine depletion is the primary treatment strategy for nephropathic cystinosis. However, poor adherence to therapy has been a major challenge resulting in poor sustained control of cystine levels, and patients consequently experience poor clinical outcomes, including kidney insufficiency leading to dialysis and kidney transplantation, muscle wasting and in some cases, premature death. Even brief interruptions in daily therapy can permit toxic accumulation of cystine, exposing tissues to renewed, progressive deterioration.
Procysbi is indicated for the management of nephropathic cystinosis patients aged 6 and older. It is contraindicated in patients hypersensitive to penicillamine.
Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases.