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FDA Approves Expanded Label for Procysbi to Treat Children Aged 2-6 Years With Nephropathic Cystinosis

Posted 20 Aug 2015 by Drugs.com

NOVATO, Calif., Aug. 17, 2015 (GLOBE NEWSWIRE) – Raptor Pharmaceutical Corp. (NASDAQ:RPTP) today announced that the U.S. Food and Drug Administration (FDA) approved the expanded use of Procysbi (cysteamine bitartrate) delayed-release capsules to treat children two to six years of age with nephropathic cystinosis. The approved supplement was based on efficacy and safety data from an ongoing long-term extension study in which a cohort of children aged 2 to 6 years were enrolled and treated with Procysbi for 12 months. Additionally, data submitted as part of this supplement supported the long-term maintenance of white blood cell cystine levels and renal function in all age groups studied during extended treatment with Procysbi. Procysbi is now approved for the treatment of nephropathic cystinosis in adult and in pediatric patients 2 years of age and older in the U.S. Please see the full ... Read more

Related support groups: Nephropathic Cystinosis, Cysteamine, Procysbi

FDA Approves Procysbi for Nephropathic Cystinosis

Posted 30 Apr 2013 by Drugs.com

April 30, 2013 – The U.S. Food and Drug Administration today approved Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis in children and adults. Procysbi was granted orphan product designation because it is intended to treat a rare disease or condition. Cystinosis is a rare genetic condition that affects an estimated 500 patients in the United States and about 3,000 patients worldwide. Fatal if not treated in early childhood, cystinosis causes a protein building block called cystine to build up in every cell of the body. The buildup of cystine causes kidney problems, which can cause the body to lose too much sugar, proteins and salts through the urine. Cystinosis may lead to slow body growth and small stature, weak bones and developing and worsening kidney failure. There are three types of cystinosis, the most severe being nephropathic cystinosis, which ... Read more

Related support groups: Cysteamine

Sigma-Tau Pharmaceuticals, Inc. Receives FDA Approval of Cystaran (cysteamine ophthalmic solution) 0.44%

Posted 9 Oct 2012 by Drugs.com

GAITHERSBURG, MD, October 04, 2012 —Sigma-Tau Pharmaceuticals, Inc. announced today that the Company has received approval from the U.S. Food & Drug Administration (FDA) for Cystaran (cysteamine ophthalmic solution) 0.44%, a topical ophthalmic therapeutic, developed in partnership with the National Institutes of Health (NIH), for the treatment of patients suffering from corneal cystine crystal accumulation as a result of cystinosis. Cystaran is designated an Orphan Drug in the U.S., and has been granted seven years of market exclusivity. Cysteamine is a cystine depleting agent which lowers the cystine content of cells in patients with cystinosis. However, when orally administered, cysteamine does not reach the cornea and is therefore ineffective in reducing the ocular effects of cystinosis. Cystaran is for topical ophthalmic use and is indicated for the treatment of corneal cystine c ... Read more

Related support groups: Eye Conditions, Cysteamine

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Further Information

Related Condition Support Groups

Nephropathic Cystinosis

Related Drug Support Groups

Cystagon, Procysbi

Cysteamine Patient Information at Drugs.com