New Treatment Approved for Nephropathic Cystinosis

Raptor Pharmaceutical announced that the FDA has approved Procysbi (cysteamine bitartrate) delayed-release capsules for the treatment of nephropathic cystinosis in patients >6 years. Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis. 

The approval is based on data from six clinical trials, including a multi-center, randomized, active-controlled Phase 3 study (N=43) in patients with nephropathic cystinosis and extension data from that trial. Procysbi demonstrated consistent cystine depletion over the full 12-hour dosing period. Sustained cystine depletion in patients may significantly delay disease progression, including kidney dysfunction, dialysis, kidney transplant, organ failure and premature death.

The FDA-approved drugs currently indicated for cystinosis include Cystagon (cysteamine bitartrate; Mylan) immediate-release tablets that were approved in 1994, and Cystaran (cysteamine; Sigma-Tau) ophthalmic solution, approved last year to treat corneal cystine crystal accumulation.

Physicians can prescribe Procysbi by calling RaptorCares at (855) 888-4004. Raptor Pharmaceutical plans to begin shipping Procysbi within 6–8 weeks. 

For more information call (855) 888-4004 or visit Raptor Pharmaceutical's website.