Studies have shown that sustained cystine depletion in patients may significantly delay disease progression, including kidney dysfunction, dialysis, kidney transplant, organ failure and premature death.
Raptor CEO and co-founder Christopher Starr said that Procysbi is the result of a decade-long patient- and physician-initiated effort to improve the treatment and lives of cystinosis patients.
Ann & Robert H. Lurie Children’s Hospital of Chicago Kidney Diseases head Craig Langman said that sustaining appropriate levels of cysteamine in the body is the key to maintaining organ function and lowering the likelihood of kidney transplantation.
"Most patients don’t take their cystinosis medication consistently as a result of severe stomach side effects and a burdensome dosing schedule," Langman added.
Clinical studies are ongoing in children less than six years of age to evaluate for the first time the safety and efficacy of Procysbi.
The FDA approval of PROCYSBI triggers the second of two $25m payments to Raptor under the terms of the HealthCare Royalty Partners (HC Royalty) $50m loan agreement.
The company is planning to launch the capsule in US by end of Q2 2013.
Image: Procysbi (cysteamine bitartrate). Photo: Courtesy of PRNewswire.