FDA Newly Approved Drug Helps Patients Reduce Cystine Levels

The Food and Drug Administration (FDA) has recently approved PROCYSBI™ (cysteamine bitartrate), a delayed release capsule for treating nephropathic cystinosis in adults and children 6 years and older. Ann and Robert H. Lurie Children’s Hospital of Chicago served as one of three United States sites for the landmark study and patients came from all over North America to be seen by lead investigator, Craig B Langman, MD, The Isaac A Abt, MD Professor of Kidney Diseases at Northwestern University Feinberg School of Medicine and Head of Kidney Diseases at Lurie Children’s.

Nephropathic cystinosis is an ultra-orphan, life-threatening metabolic disorder that causes a toxic build-up of cystine in almost all cells in the body. It affects about 500 patients in the United States and about 2000 patients world-wide. Its earliest manifestations affect the kidney, and  if undiagnosed or untreated, the affected person will suffer irreversible kidney failure by age 10, and even if a kidney transplant is performed, without life-long treatment, multi-organ failure and premature death.   

“Nephropathic cystinosis in its most common form is usually diagnosed in infancy and requires lifelong therapy,” said Langman. “Since this is a delayed release capsule, our patients only have to take it once every 12 hours instead of every six hours. Before this new medication, patients complained of side effects such as vomiting and having an “odor” about them similar to rotten eggs. That seems not to be the case with PROCYSBI in our clincical trial™.  

PROCYSBI is the culmination of early research to prove the principle of delayed release, funded by the patient advocacy group Cystinosis Research Foundation through Dr Ranjan Dohil at UCSD.  PROCYSBI is a newer formulation of the prototype drug, with both delayed release and extended action, allowing for twice daily dosing.  FDA approval was based on a New Drug Application comprising data from six clinical trials, including the first ever multi-center randomized, active-controlled Phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial, both led by Langman.1 More than 90 percent of the cystinosis population is six years or older.  At present, two additional clinical trials with PROCYSBI, led by Langman internationally, are ongoing for patients with nephropathic cystinosis

“PROCYSBI is the result of a decade-long patient- and physician-initiated effort to improve the treatment and lives of patients with nephropathic cystinosis, “said Langman.  “In addition to providing sustained control of cystine levels, this new oral medication will help our patients be more adherent with their prescription and stay the course with their treatment. That there is no need to get up in the middle of the night to take your medication is a major advantage.”  This new drug has been on the market since May 1, 2013 and physicians can now prescribe PROCYBSI.

1 A randomized controlled crossover trial with delayed-release cysteamine bitartrate in nephropathic cystinosis: effectiveness on white blood cell cystine levels and comparison of safety.  Langman CB, Greenbaum LA, Sarwal M, Grimm P, Niaudet P, Deschênes G, Cornelissen E, Morin D, Cochat P, Matossian D, Gaillard S, Bagger MJ, Rioux P.Clin J Am Soc Nephrol. 2012 Jul;7(7):1112-20. doi: 10.2215/CJN.12321211. Epub 2012 May 3. Erratum in: Clin J Am Soc Nephrol. 2013 Mar 7;8(3):468

Ann & Robert H. Lurie Children’s Hospital of Chicago, formerly Children’s Memorial Hospital, is a 23-story, state-of-the-art hospital located in downtown Chicago on the campus of its academic partner, Northwestern University Feinberg School of Medicine. Lurie Children’s is ranked as one of the nation’s top children’s hospitals in the U.S.News & World Report 2013-14 Honor Roll rankings. Lurie Children’s provides pediatric care in a setting that offers the latest benefits and innovations in medical technology, research and family-friendly design. The hospital relies on philanthropic support to care for more than 149,000 children each year.


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