Cystaran
cysteamine hydrochloride · Solution · Ophthalmic
Uses
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 )
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 )
Dosage forms and strengths
Ophthalmic solution containing 6.5 mg/mL of cysteamine hydrochloride equivalent to 4.4 mg/mL of cysteamine (0.44%). Ophthalmic solution containing 6.5 mg/mL of cysteamine hydrochloride equivalent to 4.4 mg/mL of cysteamine (0.44%). ( 3 )
Contraindications
None. None. ( 4 )
Warnings and precautions
To minimize the risk of contamination, do not touch the dropper tip to any surface. Keep bottle tightly closed when not in use. ( 5.1 ) 5.1 Contamination of Tip and Solution 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. 5.2 Benign Intracranial Hypertension 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. 5.3 Contact Lens Use 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 [see Patient Counseling Information (17) ] . 5.4 Topical Ophthalmic Use CYSTARAN is for topical ophthalmic use.
Side effects
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. 6.1 Clinical Trials 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 occurring in ≥10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects.
Use in specific populations
8.1 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 86 to 345 times the recommended human ophthalmic dose (based on body surface area) [see Data ] . CYSTARAN should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. 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 (86 to 345 times the recommended human ophthalmic dose based on a body surface area) and have revealed cysteamine bitartrate to be teratogenic. Observed teratogenic findings were intrauterine death, cleft palate, kyphosis, heart ventricular septal defects, microcephaly, exencephaly, and growth deficits. 8.2 Lactation Risk Summary There is no information regarding the presence of cysteamine in human milk, the effects on the breastfed infants, or the effects on milk production. Cysteamine administered orally is present in milk of lactating rats. It is not known whether measurable levels of cysteamine would be present in maternal milk following topical ocular administration of CYSTARAN. 8.4 Pediatric Use The safety and effectiveness of CYSTARAN (cysteamine ophthalmic solution) 0.44% have been established in pediatric patients. 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
8.1 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 86 to 345 times the recommended human ophthalmic dose (based on body surface area) [see Data ] . CYSTARAN should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. 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 (86 to 345 times the recommended human ophthalmic dose based on a body surface area) and have revealed 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
8.4 Pediatric Use The safety and effectiveness of CYSTARAN (cysteamine ophthalmic solution) 0.44% have been established in pediatric patients.
Geriatric use
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.
Description
11 DESCRIPTION CYSTARAN is a sterile ophthalmic solution containing 6.5 mg/mL of cysteamine hydrochloride, equivalent to 4.4 mg/mL of cysteamine (0.44%) as the active ingredient. Cysteamine is a cystine-depleting agent which lowers the cystine content of cells in patients with cystinosis. Molecular Formula: C 2 H 7 NS HCl Molecular Weight: 113.61 Each milliliter of CYSTARAN contains: Active: cysteamine 4.4 mg (equivalent to cysteamine hydrochloride 6.5 mg); Preservative: benzalkonium chloride 0.1 mg; Inactive Ingredients: sodium chloride, hydrochloric acid and/or sodium hydroxide (to adjust pH to 4.1-4.5), and purified water. Chemical Structure
Mechanism of action
12.1 Mechanism of Action Cysteamine acts as a cystine-depleting agent by converting cystine to cysteine and cysteine-cysteamine mixed disulfides and reduces corneal cystine crystal accumulation.
How supplied
Fifteen (15) mL of CYSTARAN (cysteamine ophthalmic solution) 0.44% is supplied in an opaque, white, low-density polyethylene (LDPE) 88 mm tall bottle with a 1.6 mm blue, silicone rubber flow-controlled dropper tip and closed with a white, high-density polyethylene (HDPE) screw cap. The bottle is foil-wrapped and stored in a carton. CYSTARAN bottles must be stored in the following conditions: NDC 54482- 020 -02 Before Opening: Store unopened bottle in freezer at -25°C to -15°C (-13°F to 5°F) in the original packaging including unopened foil. Thaw for approximately 24 hours before use. Open the carton and the foil only when starting a new bottle. After Opening: Record discard date on the bottle, which is 1 week from the day the foil and bottle were opened. Store thawed bottle at 2°C to 25°C (36°F to 77°F) for up to 1 week. The thawed bottle does not require refrigeration between use. Do not refreeze the thawed medication. Discard 1 week after the foil and bottle were opened even if there is medication left in the bottle. NDC 54482- 035 -02 Before Opening: Store unopened bottle in refrigerator at 2°C to 8°C (36°F to 46°F) in the original packaging including unopened foil. Open the carton and the foil only when starting a new bottle. After Opening: Record discard date on the bottle, which is 1 week from the day the foil and bottle were opened. During the week of use, store bottle at room temperature, 20°C to 25°C (68°F to 77°F). Discard 1 week after the foil and bottle were opened even if there is medication left in the bottle.
Storage
CYSTARAN bottles must be stored in the following conditions: NDC 54482- 020 -02 Before Opening: Store unopened bottle in freezer at -25°C to -15°C (-13°F to 5°F) in the original packaging including unopened foil. Thaw for approximately 24 hours before use. Open the carton and the foil only when starting a new bottle. After Opening: Record discard date on the bottle, which is 1 week from the day the foil and bottle were opened. Store thawed bottle at 2°C to 25°C (36°F to 77°F) for up to 1 week. The thawed bottle does not require refrigeration between use. Do not refreeze the thawed medication. Discard 1 week after the foil and bottle were opened even if there is medication left in the bottle.
Patient information
Storage of Cystaran Bottles NDC 54482- 020 -02 Patients should be advised to store unopened bottles in the freezer in the intact foil pouch and original carton. Each week, one new bottle should be removed from the freezer. Patients should be advised to allow the bottle to thaw completely (approximately 24 hours) prior to use. After the bottle is completely thawed, the patient should be advised to open the packaging (carton and foil) and record the discard date on the bottle label. The discard date is seven (7) days from the day the foil and bottle were opened. During the week of use, patients should be advised to store thawed bottle at 2°C to 25°C (36°F to 77°F) for up to 1 week. The thawed bottle does not require refrigeration between use and should not be refrozen. At the end of 1 week (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 1 week after the foil and bottle were opened. Storage of Cystaran Bottles NDC 54482- 035 -02 Patients should be advised to store unopened bottles in the refrigerator in the intact foil pouch and original carton. When starting a new bottle, patients should be advised to open the packaging (carton and foil) and record the discard date on the bottle label. The discard date is seven (7) days from the day the foil and bottle were opened. During the week of use, patients should be advised to store the bottle at room temperature 20°C to 25°C (68°F to 77°F) for up to 1 week. At the end of 1 week (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 1 week after the foil and bottle were opened. Risk of Contamination Patients should be advised not to touch the eyelid or surrounding areas with the dropper tip of the bottle. The cap should remain on the bottle when not in use. Use with Contact Lenses Patients should be advised that contact lenses should be removed prior to application of CYSTARAN. Contact lenses may be reinserted 15 minutes following CYSTARAN administration. Topical Ophthalmic Use Patients should be advised that CYSTARAN is for topical ophthalmic use.
Label text from the FDA structured product label by Leadiant Biosciences, Inc. (revised Apr 30, 2025). Long sections are shortened; the complete label is on DailyMed.
Active ingredients
- Cysteamine Hydrochloride in 2 products
Cystaran NDC products (2)
| NDC | Strength & form | Labeler | Type |
|---|---|---|---|
| 54482-020 | Cysteamine Hydrochloride 6.5 mg/mL Solution | Leadiant Biosciences, Inc. | NDA |
| 54482-035 | Cysteamine Hydrochloride 6.5 mg/mL Solution | Leadiant Biosciences, Inc. | NDA |
Cystaran recalls
- D-0034-2021 Oct 21, 2020 · Class III · Terminated
Subpotent Drug: Product testing identified a decreased level of the active ingredient, Cysteamine HCl. - D-0683-2018 Apr 25, 2018 · Class III · Terminated
Subpotent drug: Out of specification for an active ingredient cysteamine hydrochloride. - D-0550-2018 Mar 7, 2018 · Class III · Terminated
Subpotent Drug: Out of specification for an active ingredient cysteamine hydrochloride.
Frequently asked questions
What is Cystaran used for?
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 )
What are the side effects of Cystaran?
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. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely… See the full label for the complete list.
Who makes Cystaran?
Cystaran is listed by 1 labeler in the FDA NDC directory, including Leadiant Biosciences, Inc..
Has Cystaran been recalled?
The FDA enforcement database lists 3 recalls for Cystaran, most recently D-0034-2021 (class iii): Subpotent Drug: Product testing identified a decreased level of the active ingredient, Cysteamine HCl.