Raptor Pharmaceutical Corp., a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases, has launched Procysbi (cysteamine bitartrate) delayed-release capsules in the US for nephropathic cystinosis patients.

The US Food and Drug Administration (FDA) approved Procysbi on April 30, 2013 for the management of nephropathic cystinosis in adults and children ages 6 years and older. Initial prescription claims have been approved by multiple payor categories and Procysbi is being shipped to patients.

RaptorCares provides individualized services to help patients access Procysbi through education, support, extensive case management and a commitment to the principle that no eligible US patient with nephropathic cystinosis will be denied access to Procysbi based on inability to pay.

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.