Glossary
Adverse reactions: Unexpected, serious symptoms coinciding with the administration of a drug2
Amino acid: Molecules that are linked together to make proteins2
Band keratopathy: A condition in which corneal deterioration takes place, causing a line to appear across the cornea2
Benign: Allowing recovery, not life threatening2
Blepharospasm: (pronounced BLEF‑a‑row‑spa‑sum) Involuntary activity of the muscles controlling the eyelid producing more or less complete closure of the eyelids2
Cornea: The transparent covering of the eye2
Corneal abrasion: Shallow ulceration or a wearing away of the cornea2
Corneal cystine crystal: Solid cystine particles within the cornea of the eye3
Cysteamine: (pronounced sis-'tē-ǝ-mǝn) The active ingredient in CYSTARAN1
Cysteine: (pronounced 'sis-tǝ-ēn) A nonessential amino acid2
Cysteine-cysteamine mixed disulfides: An end product of cysteamine reacting with cystine crystals1
Cystine: (pronounced 'sis-tēn) An amino acid that accumulates and forms crystals in cystinosis2
Cystinosis: (pronounced sis-tǝ-'nō-sis) A rare, hereditary disease that causes a substance called cystine to build up in different organs of the body, including the kidneys and the eyes2,4
Cystinotic lysosome: A lysosome that contains an excessive amount of cystine4
Dehydration: The condition that results from excessive loss of body water2
Electrolyte: A substance that is essential to the normal function of all cells2
FDA: The US Food and Drug Administration is the government agency that is responsible for regulating food, drugs, and tobacco5
Foreign body sensation: The feeling that an irritating and abnormal substance is present2
Gene: Unit of DNA that controls the individual physical and physiologic traits inherited by offspring from their parents2
Lysosomal storage disorder: An inherited condition characterized by abnormal function of the lysosomes causing a buildup of waste2
Lysosome: The part of an animal cell that acts as the cell's waste disposal system2
Mutation: A permanent change in the DNA sequence that makes up a gene2
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
Nonnephropathic cystinosis: Typically diagnosed in adults, cystine crystals accumulate within the corneas and bone marrow. Also called benign or adult cystinosis4,6
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
Ophthalmic: Relating to the eye2
Ophthalmic solution: A sterile solution, free from foreign particles, dispensed into the eye2
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
Photophobia: Abnormal visual intolerance to light2
Prescribing Information: Also referred to as the package insert or PI. This document provides the FDA-approved medical information for a drug
Recessive gene: A gene that produces an effect in offspring only when it is passed on by both parents2
Recessive genetic disease: A disorder that is caused by the inheritance of 2 otherwise inactive or recessive genes (see definition for recessive gene)2
Renal tubular Fanconi syndrome: A condition that
makes the kidneys unable to reabsorb nutrients and
minerals that are
lost in the urine6
Rickets: A condition that leads to softening and weakening of bones. In cystinosis, this is caused by the increased loss of phosphorus through the kidneys.2
References: 1. CYSTARAN [prescribing information]. Gaithersburg, MD: Leadiant Biosciences, 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.
Indication:
CYSTARAN is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.
Important Safety Information:
The most frequently reported ocular adverse reactions occurring in ≥10% of patients were sensitivity to light, redness, eye pain/irritation, headache, and visual field defects.