The FDA has approved cysteamine bitartrate (Procysbi) for the treatment of nephropathic cystinosis in patients aged six years and older.
In a Phase 3 study, cysteamine bitartrate demonstrated a consistent rate of cystine depletion in a 12-hour dosing period, which can be beneficial in significantly delaying disease progression.
The drug is manufactured by Raptor Pharmaceuticals Corp. based in Novato, California.
“Sustaining appropriate levels of cysteamine in the body is the key to maintaining organ function and lowering the likelihood of kidney transplantation,” said Craig Langman, MD, The Isaac A. Abt, MD Professor of Kidney Diseases Feinberg School of Medicine and Head of Kidney Diseases at Ann & Robert H. Lurie Children’s Hospital of Chicago. “Most patients don’t take their cystinosis medication consistently as a result of severe stomach side effects and a burdensome dosing schedule. In addition to providing sustained control of cystine levels, PROCYSBI’s dosing schedule and side effect profile may help patients stay the course with their treatment.”