Rare Disease Report: Raptor’s Cysteamine Capsule Now Approved for Children

September 7, 2015

Children with nephropathic cystinosis can now receive delayed-release cysteamine tablets. Raptor Pharmaceutical Corp announced that the U.S. Food and Drug Administration (FDA) approved the expanded use of Procysbi (cysteamine bitartrate) delayed-release capsules to treat children 2 to 6 years of age with nephropathic cystinosis. The orphan drug was initially approved in 2013 for adults.

The label expansion was based on data from an ongoing long-term extension study in which a cohort of children aged 2 to 6 years were enrolled and treated with the cysteamine capsules for 12 months. With the label extension, all patients with nephropathic cystinosis 2 years and older can receive the drug.

Craig Langman, MD, head of kidney diseases at Ann & Robert H. Lurie Children’s Hospital of Chicago and the lead investigator on the extension trial said, “Strict adherence to cystine depleting therapy from as early an age as possible is critical to maintaining kidney function and leading longer, healthier lives for patients with cystinosis.” Dr Langman added, “The data in the two to six year old group confirm the maintenance of stable kidney function over time. Not only will this help these young patients with cystinosis, but the 12 hour dosing schedule for Procysbi could be a significant benefit to the caregivers of these young children.”

“Caregivers of younger individuals with cystinosis will now have another treatment option to discuss with their medical professional teams,” stated Jeff Larimore, President of the Cystinosis Research Network. “Every opportunity provided to improve treatment adherence is applauded by the cystinosis community.”

About Cystinosis

Cystinosis is an autosomal recessive, metabolic disease in which the amino acid cystine accumulates in various organs of the body such as the kidney, eye, muscle, pancreas, and brain.

There are three clinical forms of cystinosis. Infantile (or nephropathic) cystinosis; late-onset cystinosis; and benign cystinosis. Of these, nephropathic cystinosis is the most severe and usually diagnosed between 6 and 18 months of age.  Common symptoms include excessive thirst and urination, failure to thrive, rickets, and episodes of dehydration. Children with cystinosis can also develop crystals in their eyes (after one year of age) and without specific treatment, develop end-stage renal failure may occur at approximately 9 years of age. Kidney transplantation has proven very helpful in patients with cystinosis.

There are an estimated 500 patients living in the United States with cystinosis, and 2,000 worldwide.

Orphan drugs approved for cystinosis include

• Cystagon (cysteamine) and Procysbi (extended release cysteamine)  are approved to  nephropathic cystinosis.
• Cystaran (cysteamine eye drops) to treat corneal cysteine crystal accumulation in patients with cystinosis.


Raptor Pharmaceutical Receives FDA Approval of Expanded Label for PROCYSBI(R) to Treat Children Aged 2-6 Years With Nephropathic Cystinosis [press release]. Novato, CA: Raptor Pharmaceuticals Corp; August 17, 2015.

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