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NOVATO, Calif. — The Food and Drug Administration has given special designation to a drug made by Raptor Pharmaceuticals for a rare metabolic disease.
Raptor said the FDA granted it orphan product exclusivity for Procysbi (cysteamine bitartrate) delayed-release capsules for managing nephropathic cystinosis in patients aged 6 and older. Nephropathic cystinosis is a rare, life-threatening lysosomal storage disorder that causes toxic accumulation of an amino acid called cystine in the body's cells, tissues and organs, leading to progressive and irreversible tissue damage and organ failure. An estimated 500 patients in the United States have the disease.
"This decision was the outcome of a productive and cooperative process with the FDA," Raptor chief business officer Thomas Daley said. "We believe the issuance of orphan drug exclusivity highlights an appreciation that Procysbi is an important new therapeutic option for nephropathic cystinosis patients."
The drug was approved in April, and the exclusivity period lasts for seven years. Under normal circumstances, the FDA gives a pharmaceutical drug an exclusivity period of five years, meaning generic drug makers must wait until the exclusivity period has expired before they may file for approval of a generic version.