FDA Expands Use of Rare Kidney Disease Treatment

Nephropathic cystinosis drug Procysbi now available in delayed-release capsules for young children.

Young children with a rare kidney condition will soon have a new treatment option.

The FDA has expanded the use of Raptor Pharmaceutical’s cysteamine bitartrate (Procysbi) delayed-release capsules to treat nephropathic cystinosis in children ages 2 to 6.

"Strict adherence to cysteine-depleting therapy from as early an age as possible is critical to maintaining kidney function and leading longer, healthier lives for patients with cystinosis," noted Craig Langman, MD, head of kidney diseases at Ann & Robert H. Lurie Children's Hospital of Chicago, in a press release. “Not only will [Procysbi] help these young patients with cystinosis, but the 12-hour dosing schedule…could be a significant benefit to the caregivers of these young children."

Procysbi’s expanded approval was based on data from an extension study that demonstrated long-term maintenance of white blood cell cystine levels and renal function in children with nephropathic cystinosis taking the drug for 12 months.

Now, Procysbi is approved for the treatment of nephropathic cystinosis in adult and pediatric patients ages 2 years and older. Nephropathic cystinosis comprises 95% of diagnosed 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.

The most common side effects associated with Procysbi are vomiting, nausea, diarrhea, and abdominal pain. The drug is contraindicated in patients with a hypersensitivity to cysteamine or penicillamine.