Data from FDA - Curated by Marshall Pearce - Last updated 01 January 2018

Indication(s)

1 INDICATIONS AND USAGE CYSTARAN is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. CYSTARAN is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. (1)

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Advisory information

contraindications
4 CONTRAINDICATIONS None. None. (4)
Adverse reactions
6 ADVERSE REACTIONS Clinical Studies Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described below reflect exposure in controlled clinical trials of six months to 19 years duration in approximately 300 patients. The most frequently reported ocular adverse reactions occuring in ≥10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects. The most common adverse reactions (incidence approximately 10% or greater) are sensitivity to light, redness, eye pain/irritation, headache and visual field defects. (6) To report SUSPECTED ADVERSE REACTIONS, contact Leadiant Biosciences, Inc. at 1-888-393-4584 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Usage information

Dosing and administration
2 DOSAGE AND ADMINISTRATION Instill one drop of CYSTARAN in each eye, every waking hour. Do not touch dropper tip to any surface, as this may contaminate the solution. Discard after 1 week of use. Instill one drop of CYSTARAN in each eye, every waking hour. (2)
Use in special populations
8 USE IN SPECIFIC POPULATIONS 8.1 Pregnancy There are no adequate and well-controlled studies of ophthalmic cysteamine in pregnant women. CYSTARAN should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Teratogenic Effects: Pregnancy Category C. Teratology studies have been performed in rats at oral doses in a range of 37.5 mg/kg/day to 150 mg/kg/day (about 0.2 to 0.7 times the recommended human maintenance dose on a body surface basis) and have revealed cysteamine bitartrate to be teratogenic. Observed teratogenic findings were cleft palate, kyphosis, heart ventricular septal defects, microcephaly, and exencephaly. Nonteratogenic Effects: Cysteamine was fetotoxic, resulting in intrauterine death and growth retardation in rats at oral doses of 0.2 to 0.7 times the recommended human maintenance dose on a body surface basis. 8.3 Nursing Mothers It is not known whether oral cysteamine is excreted in human milk. Because many drugs are excreted in human milk and because of the manifested potential of cysteamine for developmental toxicity in suckling rat pups when it was administered to their lactating mothers at an oral dose of 375 mg/kg/day (2,250 mg/m2/day, 1.7 times the recommended human dose based on body surface area), a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother. The incremental increase in systemic cysteamine levels derived from drug applied topically to the eye in patients treated with oral cysteamine is negligible. 8.4 Pediatric Use The safety and effectiveness of CYSTARAN (cysteamine ophthalmic solution) 0.44% have been established. 8.5 Geriatric Use When the clinical studies with CYSTARAN were conducted, the reduced life expectancy from cystinosis did not make it possible to include patients in the geriatric age range. 8.6 Renal Impairment The effect of renal impairment on the pharmacokinetics of cysteamine following ophthalmic administration of cysteamine ophthalmic solution has not been evaluated because ophthalmic exposure compared to systemic exposure is negligible. The majority of the patients in the ophthalmic clinical studies are assumed to have had some degree of renal impairment due to their underlying systemic disease. The total daily ophthalmic dose is less than 2% of the recommended oral daily dose of cysteamine; thus, the systemic exposure following ophthalmic administration is expected to be negligible compared to oral administration.
Pregnancy and lactation
8.3 Nursing Mothers It is not known whether oral cysteamine is excreted in human milk. Because many drugs are excreted in human milk and because of the manifested potential of cysteamine for developmental toxicity in suckling rat pups when it was administered to their lactating mothers at an oral dose of 375 mg/kg/day (2,250 mg/m2/day, 1.7 times the recommended human dose based on body surface area), a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother. The incremental increase in systemic cysteamine levels derived from drug applied topically to the eye in patients treated with oral cysteamine is negligible.

More information

Category Value
Authorisation number NDA200740
Agency product number IF1B771SVB
Orphan designation No
Product NDC 54482-020
Date Last Revised 31-07-2017
Type HUMAN PRESCRIPTION DRUG
RXCUI 1313780
Marketing authorisation holder Leadiant Biosciences, Inc.