Nephropathic cystinosis: A genetic metabolic disease that causes an amino acid, cystine, to accumulate in various organs of the body. In untreated children it is characterized by renal tubular Fanconi syndrome, poor growth, hypophosphatemic rickets, impaired glomerular function, and accumulation of cystine crystals in almost all cells, leading to tissue destruction2,6
Ocular cystinosis: A term used to refer to cystinosis affecting only the eye. This is sometimes called benign or adult cystinosis; however, the NIH has proposed this terminology no longer be used. The preferred term is nonnephropathic cystinosis6,7
Orphan drug: A drug that meets FDA criteria, such as treating a disease or condition that is rare. Any disease or condition that affects fewer than 200,000 people in the United States is considered rare7
References: 1. CYSTARAN [prescribing information]. Gaithersburg, MD: Sigma-Tau Pharmaceuticals, Inc; 2012. 2. Dorland’s Online Medical Dictionary. Available at: http://www.dorlands.com. Accessed March 13, 2013. 3. Kaiser-Kupfer MI, Fujikawa L, Kuwabara T. Removal of corneal crystals by topical cysteamine in nephropathic cystinosis. N Engl J Med. 1987;316:1775-1779. 4. Gahl WA, Kuehl EM, Iwata F, Lindblad A, Kaiser-Kupfer MI. Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops. Mol Genet Metab. 2000;71:100-120. 5. U.S. Food and Drug Administration Web site. About FDA: FDA Fundamentals. Available at: http://www.fda.gov/AboutFDA/Transparency/ Basics/ucm192695.htm. Accessed March 13, 2013. 6. Nesterova G, Gahl WA. Cystinosis. In: Pagon RA, Bird TC, Dolan CR, Stephen K, eds. GeneReviews [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2001. Available at: http://www.ncbi.nlm.nih.gov/books/NBK1400. Accessed March 13, 2013. 7. U.S. Food and Drug Administration Web site. Orphan Drug Act. Available at: http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/ OrphanDrugAct/default.htm. Accessed March 13, 2013.
CYSTARAN (CYSTEAMINE OPHTHALMIC SOLUTION) 0.44% STERILE is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.
Important Safety Information:
To minimize contaminating the dropper tip and solution, care should be taken not to touch the eyelids or surrounding areas with the dropper tip of the bottle. Keep bottle tightly closed when not in use.
There have been reports of benign intracranial hypertension (or pseudotumor cerebri) associated with oral cysteamine treatment that has resolved with the addition of diuretic therapy. There have also been reports associated with ophthalmic use of cysteamine; however, all of these patients were on concurrent oral cysteamine.
CYSTARAN contains benzalkonium chloride, which may be absorbed by soft contact lenses. Contact lenses should be removed prior to application of solution and may be reinserted 15 minutes following its administration.
CYSTARAN is for topical ophthalmic use only.
The most frequently reported ocular adverse reactions occurring in ≥ 10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects.