The Cystinosis Advocate
Volume 5, Issue 2 P a g e 1 The Cystinosis Advocate Volume 5, Issue 2 Fall/Winter, 2012 Inside this issue: Sigma-Tau Pharmaceuticals, Inc. Receives President’s Message 2-3 FDA Approval of CYSTARAN™ Cystaran FAQ 4-5 GAITHERSBURG, MD, October 4, 2012—Sigma-Tau Pharmaceuticals, Inc. announced today that the Company has received approval from the U.S. Food & Drug Mexico Symposium 6-7 Administration (“FDA”) for CYSTARAN™ (cysteamine ophthalmic solution) 0.44%, a topical Financial Update 8-9 ophthalmic therapeutic, developed in partnership with the National Institutes of Health Family Support Update 10 (“NIH”), for the treatment of patients suffering from corneal cysteine crystal accumulation as a result of cystinosis. CYSTARAN™ is designated an Orphan Drug in the U.S. and has Family Story 10 been granted seven years of market exclusivity. Midwest Gathering 11 Cysteamine is a cysteine-depleting agent which lowers the cysteine content of cells in Research Update 12 patients with cystinosis. However, when New Studies Enrolling 13 orally administered, cysteamine does Celebration of Science 14-15 not reach the cornea and is therefore ineffective in reducing the ocular effects Rare Disease Conf. 16 of cystinosis. CYSTARAN™ is for topical Awareness Bracelets 17 ophthalmic use and is indicated for the RaptorCares 18-19 treatment of corneal cystine crystal accumulation in patients with cystinosis. Education Update 20 As a result, CYSTARAN™ now represents the only FDA-approved ophthalmic treatment for NORD Update 21 this condition. Development Update 22-23 “As a Company dedicated to the development and commercialization of novel therapies Utah Golf Fundraiser 24-25 that address the unmet medical needs of a wide range of rare disease patients, we are Family Story 26-27 delighted to announce the approval of CYSTARAN™,” noted Dave Lemus, Chief Operating (continued on page 4) Halloween Fundraiser 28-29 CRN Family Conference: Heroes Among Us July 18-20, 2013 in Washington, D.C.
[Show full text]