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What is CYSTARAN®?

boy in forest looking at camera

What is CYSTARAN®?

boy in forest looking at camera

What is
CYSTARAN®?

What is CYSTARAN?

CYSTARAN is an eyedrop medication intended to treat the buildup of cystine crystals in the corneas of adults and children who have cystinosis. It was approved by the FDA in 2012 as an orphan drug.

the letters f y and then eye that blinks
An orphan drug is a medication that treats a rare condition affecting under 200,000 people in the United States. Learn more.

CYSTARAN is a topical medication, which means that you put it directly on the affected area—in this case, the eyes. That’s different from an oral medication, which you take by mouth. If you have cystinosis, you may be prescribed both an oral and a topical medication.

How does CYSTARAN work?

Cysteamine, the active ingredient in CYSTARAN, works to help decrease the accumulation of cystine crystals in the body’s tissues.

Cysteamine binds to and changes cystine. The bound substance can be removed from the lysosomes of the body’s cells without a need for cystinosin and is not harmful to the eye.

Lysosome Affected by Cystinosis

Lysosome Affected by Cystinosis
After Taking Cysteamine

animation of eye crystal accumulation cystinosis
how cystaran works to treat eye cystinosis

The use of CYSTARAN has been demonstrated to be effective at reducing corneal crystals.

How does CYSTARAN work?

Cysteamine, the active ingredient in CYSTARAN, works to help decrease the accumulation of cystine crystals in the body’s tissues.

Cysteamine binds to and changes cystine. The bound substance can be removed from the lysosomes of the body’s cells without a need for cystinosin and is not harmful to the eye.

Lysosome Affected by Cystinosis

animation of eye crystal accumulation cystinosis

Lysosome Affected by Cystinosis After Taking Cysteamine

how cystaran works to treat eye cystinosis

The use of CYSTARAN has been demonstrated to be effective at reducing corneal crystals.

See examples of Treatment with CYSTARAN*

eye cystinosis patients' corneas before and after treatment
eye cystinosis patients' corneas before and after treatment
eye cystinosis patients' corneas before and after treatment

*Corneal slit-lamp photographs of patients treated with CYSTARAN. These examples represent patients who responded to treatment and in subsequent follow up appointments. Duration of therapy varied from 8-41 months. Response may vary from patient to patient.

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How do I store and take CYSTARAN?

How to Store CYSTARAN

refrigerator icon

How do I store and take CYSTARAN?

How to Store CYSTARAN

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Store bottles in the freezer in the original carton.

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Each week, remove one new bottle from the freezer.

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Allow the bottle to thaw completely (approximately 24 hours) prior to use.

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After the bottle is completely thawed, record the discard date on the bottle label. The discard date is seven (7) days from the day the bottle is thawed.

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Store the thawed bottle at no greater than room temperature for up to 1 week. No refrigeration required after thawing. Do not refreeze the bottle.

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Remember to discard the bottle at the end of 1 week (7 days). There may be medication left in the bottle; however, you must discard it because the medication is only stable for 1 week after thawing.

refrigerator icon with check mark

Store bottles in the freezer in the original carton.

bottle with arrow pointing to calendar icon

Each week, remove one new bottle from the freezer.

ice crystal thawing icon

Allow the bottle to thaw completely (approximately 24 hours) prior to use.

bottle with arrow to trash can and calendar

After the bottle is completely thawed, record the discard date on the bottle label. The discard date is seven (7) days from the day the bottle is thawed.

thermometer with checkmark

Store the thawed bottle at no greater than room temperature for up to 1 week. No refrigeration required after thawing. Do not refreeze the bottle.

trash can icon

Remember to discard the bottle at the end of 1 week (7 days). There may be medication left in the bottle; however, you must discard it because the medication is only stable for 1 week after thawing.

the letters f y and then eye that blinks
There’s no need to store CYSTARAN in the refrigerator during the week that you’re using it. It can be safely stored at room temperature after it’s removed from the freezer.

How to Take CYSTARAN

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Always wash your hands thoroughly with soap and water before administering eye drops.
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Instill one drop of CYSTARAN in each eye, every waking hour

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Do not touch dropper tip to any surface, as this may contaminate the solution

calendar with arrow for week

Discard after
1 week of use

trash can icon

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 thawing

How to Take CYSTARAN

hands washing icon
Always wash your hands thoroughly with soap and water before administering eye drops.
bottle with droplet coming out and clock

Instill one drop of CYSTARAN in each eye, every waking hour

don't touch tip of bottle icon

Do not touch dropper tip to any surface, as this may contaminate the solution

calendar with arrow for week

Discard after 1 week of use

trash can icon

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 thawing

the letters f y and then eye that blinks
Early care from an ophthalmologist (an eye specialist) can help cystinosis patients avoid eye problems that may impact their quality of life.

Where do I order CYSTARAN?

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CYSTARAN is ONLY available from AllianceRx Walgreens Prime Pharmacy.

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Your medication will be shipped directly to your home.

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To order, call 1-877-534-9627

Friendly associates exclusively available to help CYSTARAN patients will be happy to assist you Mon–Fri, 8AM–7PM EST.

telephone icon

To order, call 1-877-534-9627

Friendly associates exclusively available to help CYSTARAN patients will be happy to assist you Mon–Fri, 8AM–7PM EST.

Patient Support Services

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Leadiant Biosciences, Inc. is committed to helping patients and families access our products, including CYSTARAN. Learn what advocacy and assistance services are available to cystinosis patients here.

Frequently Asked Questions (FAQs)

If a disease affects fewer than 200,000 people in the U.S., it’s considered an orphan disease according to the FDA’s standards. It’s estimated that cystinosis only affects around 600 children and adults in the U.S. A drug approved to treat an orphan disease is called an orphan drug. CYSTARAN was approved as an orphan drug in 2012. You can find more information about the FDA’s orphan drug program for rare diseases here.

Your doctor will inform you of your condition or diagnosis. You or your child may have what’s called nephropathic cystinosis (which means cystinosis that involves the kidneys). Nephropathic cystinosis is typically noticed by doctors very early in a patient’s life.

The most commonly reported type of nephropathic cystinosis shows up in infancy. Infants and children with nephropathic cystinosis will show a specific set of symptoms (see below). Doctors can diagnose nephropathic cystinosis by measuring the amount of cystine in the blood.

Non-nephropathic cystinosis (also called ocular cystinosis) is usually diagnosed much later in life when corneal cystine crystals are detected in an eye exam.

Early signs and symptoms include:

cystinosis symptoms image

Non-nephropathic (ocular) cystinosis is a type of cystinosis that only affects the eyes. It is sometimes called benign or adult cystinosis since it does not cause life-threatening symptoms or kidney disease. The only symptom of non-nephropathic cystinosis is corneal crystal accumulation. Non-nephropathic cystinosis is typically noticed later in life than the other forms of cystinosis. Eye doctors can diagnose non-nephropathic cystinosis if crystals are seen during an eye exam.

Cystine crystals are formed when lots of cystine (an amino acid) begins to accumulate in one place. The structures that form when cystine accumulates are called crystals because of the specific way the molecules of cystine arrange themselves.

It is best to talk to an ophthalmologist right away. An ophthalmologist is an eye specialist who should be able to diagnose cystine crystals in your eyes.

After consulting with your physician you may receive a topical cystine depleting treatment, like CYSTARAN. CYSTARAN is FDA-approved to treat the accumulation of corneal cystine crystals in cystinosis patients. It is an eyedrop treatment that you apply directly on the eye.

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IMPORTANT SAFETY INFORMATION

What is CYSTARAN?

CYSTARAN (cysteamine ophthalmic solution) 0.44% is an eyedrop medication used to treat cystine crystal accumulation in the corneas of patients who have cystinosis.

What is the most important safety information I should know about CYSTARAN?

  • To help prevent contamination of the dropper tip and eyedrop medication, try to make sure that CYSTARAN is dropped directly onto the eye without touching it. Try not to touch the eyelids or surrounding areas with the dropper tip of the bottle when you are using CYSTARAN. Keep the bottle tightly closed when not in use.
  • CYSTARAN contains an ingredient called benzalkonium chloride which can be absorbed by soft contact lenses. Remove contact lenses before using CYSTARAN eyedrops and wait at least 15 minutes before reinserting them.
  • CYSTARAN should only be used as an eyedrop medication.

What are the side effects of CYSTARAN?

  • The most common side effects of CYSTARAN, which have occurred in at least 10% of people using the medication, were sensitivity to light, eye redness, eye pain and irritation, and headache.

The risk information provided here is not comprehensive. To learn more, talk to your healthcare provider or pharmacist about CYSTARAN. The full FDA-approved product labeling can be found at www.cystaran.com.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call FDA at 1-800-FDA-1088.

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IMPORTANT SAFETY INFORMATION

What is CYSTARAN?

CYSTARAN (cysteamine ophthalmic solution) 0.44% is an eyedrop medication used to treat cystine crystal accumulation in the corneas of patients who have cystinosis.