Cystadrops Drug Information

is a cystine-depleting agent indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis. CYSTADROPS is a cystine-depleting agent indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis.

Dosage Information Instill one drop of

CYSTADROPS in each eye, 4 times a day during waking hours. Do not touch dropper tip to the eyelids, surrounding areas, or any surface, as this may contaminate the solution. In case of concomitant therapy with other topical ocular products, an interval of 10 minutes should be allowed between successive applications.

Eye ointments should be administered last. If the patient misses an instillation, the patient should be told to administer a dose as soon as feasible and then continue the treatment with the next scheduled instillation. Discard bottle 7 days after first opening.

Preparation for

Administration Patients should be advised to store new unopened CYSTADROPS bottles in the refrigerator in the original carton between 36°F to 46°F (2°C to 8°C). Each week, one new bottle should be removed from the refrigerator. Patients are to write the date the bottle was opened in the space on the carton. After first opening, store opened CYSTADROPS at room temperature between 68°F to 77°F (20°C to 25°C). Do not refrigerate after opening.

Patients are to wash their hands carefully in order to avoid microbiological contamination of the content in the bottle. Remove the green protective cap (see Figure A ). Remove the metal seal (see Figure B ). Remove the gray stopper (see Figure C ) from the bottle. Do not touch the opening of the bottle after removing the gray stopper.

Take the dropper out of its packaging, without touching the end intended to be attached to the bottle, attach it (see Figure D ) to the bottle and do not remove it. Patients should be advised not to lose the small white cap that comes on the top of the dropper (see Figure E ). Keep the small white cap tightly closed when not in use. Instill one drop of CYSTADROPS in each eye, 4 times a day during waking hours.

At the end of 7 days, patients should discard the bottle. There may be medication left in the bottle; however, the bottle must be discarded by the patient because the medication is only stable for 7 days after first opening.

Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in 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 most common adverse reactions (≥ 10%) reported during clinical trials were eye pain, vision blurred, eye irritation, ocular hyperaemia, instillation site discomfort, eye pruritus, lacrimation increased, and ocular deposits.

Pregnancy Risk Summary There are no adequate and well-controlled studies of ophthalmic cysteamine in pregnant women to inform any drug associated risks. Oral administration of cysteamine to pregnant rats throughout the period of organogenesis was teratogenic at doses 240 to 960 times the recommended human ophthalmic dose (based on body surface area) . CYSTADROPS should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal data Teratology studies have been performed in rats at oral doses in the range of 37.5 mg/kg/day to 150 mg/kg/day (240 to 960 times the recommended human ophthalmic dose based on body surface area) and have shown cysteamine bitartrate to be teratogenic. Observed teratogenic findings were intrauterine death, cleft palate, kyphosis, heart ventricular septal defects, microcephaly, exencephaly, and growth deficits.

Pediatric Use The safety and effectiveness of CYSTADROPS has been established in pediatric patients. Use of CYSTADROPS is supported by adequate and well controlled trials in pediatric patients and additional experience supporting the safety of CYSTADROPS.