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Prescribing Information


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How is nephropathic cystinosis treated?

Treatment is focused on managing the symptoms, since there is no cure.2

An oral treatment called cysteamine can help3:

  • Lower the amount of cystine in the cells
  • Improve growth in children and help them develop more normally
  • Keep the patient’s own kidneys functioning longer

It is important for people whose kidneys are affected to drink lots of fluids, to prevent dehydration. Electrolytes, vitamins, and minerals may be added to the diet to keep them in balance and replace those that have been lost.4

How are corneal cystine crystals treated?

The treatment for corneal cystine crystals is the same whether it’s nonnephropathic cystinosis or nephropathic cystinosis. Because oral cysteamine does not reach the corneas, ophthalmic treatment is needed.5 CYSTARAN is an FDA-approved ophthalmic treatment for this condition. In clinical studies, CYSTARAN depleted cystine crystals within the cornea. It may have also helped to relieve the sensitivity to light known as photophobia.1,6

How do you use CYSTARAN eye drops?

CYSTARAN should be given as 1 drop in each eye every waking hour, unless your doctor gives you other instructions. To avoid contamination, do not touch the dropper tip to any surface. Keep bottle tightly closed when not in use.1

What is the proper storage and handling of CYSTARAN eye drops?

CYSTARAN will arrive frozen. Thaw a bottle for approximately 24 hours before use. Do not heat. Write the discard date on the bottle label. The discard date is seven (7) days from the day the bottle is thawed. Store thawed CYSTARAN drops at 2°–25° C (36°–77° F) for up to 1 week. Do not refreeze. Discard the bottle after 7 days, even if there is still medication in it. CYSTARAN solution is only stable for 1 week.1

What if patients wear contact lenses?

Contact lenses should be removed before using CYSTARAN drops. Contact lenses may be reinserted 15 minutes following CYSTARAN administration.1

References: 1. CYSTARAN [prescribing information]. Gaithersburg, MD: Leadiant Biosciences, Inc.; 2020. 2. Cystinosis Research Foundation Web site. What is cystinosis. Available at: Accessed June 15, 2020. 3. Cystinosis Research Network Web site. Cystinosis symptoms & treatment. Available at: Accessed March 13, 2013. 4. Elenberg E. eMedicine Pediatrics: General Medicine Web site. Cystinosis. Available at: Accessed March 13, 2013. 5. 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. 6. Data on file. Leadiant Biosciences, Inc.


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.