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Prescribing Information

 

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Frequently Asked Questions

What is cystinosis?

Cystinosis is a rare genetic lysosomal storage disorder that causes abnormal accumulations of the amino acid cystine. Accumulated cystine can form crystals in the patient’s major organs. The buildup of cystine crystals in the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells slowly devastates the organs.1 It is estimated that there are 500 patients in the United States and 2000 individuals worldwide with cystinosis.1,2

What is an orphan drug? Is CYSTARAN (cysteamine ophthalmic solution) an orphan drug?

  • The definition of an orphan drug is, in part, that it treats a disease or condition that is rare. Any disease or condition that affects fewer than 200,000 people in the United States is considered rare3
  • There are about 500 people in the United States with cystinosis, so it fits the classification of a rare disease. Cysteamine hydrochloride was given the Orphan Drug Designation in August 19972,4

What is CYSTARAN and what is it indicated for?

CYSTARAN is an ophthalmic solution indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.5 Please see full Prescribing Information.

How does CYSTARAN work?

Cysteamine acts as a cystine-depleting agent. It enters the cystinotic lysosome and reacts with the cystine. The result is cysteine and cysteine-cysteamine mixed disulfides. These molecules are able to exit the lysosome via a transport system that is normal in patients with cystinosis. This ultimately results in reduction of the corneal cystine crystals.5,6

Who should use CYSTARAN?

Patients with cystinosis. Patients should always consult with their treating physicians before beginning therapy.

How can I get a prescription for CYSTARAN?

Please speak with your treating physician.

When was CYSTARAN FDA approved?

October 2, 2012.

Where can I get CYSTARAN?

Your CYSTARAN prescription will be dispensed by Walgreens Specialty Pharmacy (1‑877‑534‑9627). Walgreens will ship the product directly to patients’ homes.

What are the side effects?

The most common adverse reactions are sensitivity to light, redness, eye pain/irritation, headache, and visual field defects.6 Please see the full Prescribing Information for more information.

How do I find out more information about reimbursement and assistance programs?

  • We believe CYSTARAN will be fully reimbursed by medical insurance and prescription drug plans. Anyone with questions about reimbursement should call the CYSTARAN Hotline at 1‑877‑534‑9627
  • For those patients without insurance, Sigma-Tau has created the Sigma-Tau Patient Assistance Program, which provides access to CYSTARAN eye drops for patients who are otherwise unable to afford it
  • Our goal is to ensure that CYSTARAN is available to all patients who need it

Will product availability be restricted?

Our goal is to ensure that CYSTARAN is available for all patients who require it.

Can I still use a compounding pharmacy?

You should discuss this with your treating physician.

What should I do with the drops I already have?

Continue to use the drops as directed by your physician.

How will my CYSTARAN prescription be shipped?

Each prescription of CYSTARAN will be shipped next day in a specially designed dry ice unit that is qualified for cold storage for 72 hours, ensuring that the drug maintains its optimal -25º to -15º C (-13º to 5º F) temperature. There is a temperature indicator in the shipping package that will alert users in the event the product quality could be jeopardized.

How long should the bottle be out of the freezer before use?

Prior to opening a new bottle, you should allow the bottle to thaw at approximately 2°–25° C (36°–77° F) 24 hours before use. The bottle should be completely thawed after 24 hours. Do not heat bottle or its contents.6 Please see the full Prescribing Information for more information.

How am I supposed to take the drops?            

CYSTARAN should be given as 1 drop in each eye every waking hour, unless your doctor gives you other instructions. To minimize contaminating the dropper tip and solution, care should be taken no to touch the eyelids or surrounding areas with the dropper tip. Keep bottle tightly closed when not in use.6

What if patients wear contact lenses?

Cystaran contains benzalkonium chloride, which may be absorbed by soft contact lenses. Contact lenses should be removed before using CYSTARAN drops. Contact lenses may be inserted 15 minutes following CYSTARAN administration.6

How the drops should be stored:

When unopened: In the freezer
When opened: 2°–25° C (36°–77° F)
At school: Discuss with your local school the options for your child, as rules for each school district vary.

Please see the full Prescribing Information for more information.

How often should I change the bottles?

You should discard the bottle and any remaining solution 1 week from the day you first open it. After opening the bottle for the first time, write the “discard date” on the bottle — 1 week from the date of the first opening.6

References: 1. Cystinosis Research Network Web site. Cystinosis symptoms & treatment. Available at: http://www.cystinosis.org/symptoms-treatments. Accessed March 13, 2013. 2. The Scripps Research Institute Web site. Rare genetic disease successfully reversed using stem cell transplantation [press release]. La Jolla, CA: The Scripps Research Institute; September 17, 2009. Available at: http://www.scripps.edu/news/press/ 091709.html. Accessed March 13, 2013. 3. U.S. Food and Drug Administration Web site. Orphan Drug Act. Available at: http://www.fda.gov/RegulatoryInformation/Legislation/ FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/OrphanDrugAct/default.htm. Accessed March 13, 2013. 4. U.S. Food and Drug Administration Web site. FDA Application Search Orphan Drug Designations and Approvals. Available at: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. 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. CYSTARAN [prescribing information]. Gaithersburg, MD: Sigma-Tau Pharmaceuticals, Inc; 2012.

Indication:

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.