Cysteamine bitartrate

What is Cysteamine bitartrate?

• Cysteamine bitartrate (PROCYSBI) is a cystine-depleting agent used to treat nephropathic cystinosis.

What are the uses of this medicine?

• This medicine is used to treat nephropathic cystinosis in adults and children 1 year of age and older.

How does this medicine work?

• Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.

Who Should Not Use this medicine ?

This medicine cannot be used in patients:

• with a serious hypersensitivity reaction, including anaphylaxis, to penicillamine or cysteamine.

What drug interactions can this medicine cause?

• Drugs that increase the gastric pH (e.g., medications containing bicarbonate or carbonate) may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration.
• Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release. Therefore, do not consume alcoholic beverages during treatment with PROCYSBI.

Is this medicine FDA approved?

• FDA approved this drug in the year of 2013.

How should this medicine be used?

Recommended Dosage: Cysteamine-naïve Patients:

• Start PROCYSBI at a fraction of the maintenance dosage.
• Patients 1 year to less than 6 years: Gradually increase the dosage, allowing a minimum of 2 weeks between adjustments.
• Patients 6 years of age and older: Gradually increase the dosage over 4 to 6 weeks until the maintenance dosage is achieved.

Switching from Immediate-release Cysteamine to PROCYSBI:

• Start with a total daily dose of PROCYSBI equal to the previous total daily dose of immediate-release cysteamine bitartrate.

Dose Titration:

• Adjust dose to achieve a therapeutic target white blood cell (WBC) cystine concentration.
• If a dose adjustment is required, increase the dosage by 10%. The maximum dosage is 1.95 grams/m2 per day.
• If adverse reactions occur, decrease the dosage. Some patients may be unable to achieve their therapeutic target.

Administration

• Take PROCYSBI exactly as your doctor tells you to.
• Your doctor may start you on a low dose of PROCYSBI and slowly increase your dose to help avoid side effects, especially if you have not taken a medicine that contains cysteamine bitartrate before.
• Do not change your dose of PROCYSBI unless your doctor tells you to.
• PROCYSBI is taken 2 times each day, every 12 hours.
• Take PROCYSBI the same way each time, either without eating or with a small amount of food, as follows:
• If you take PROCYSBI without eating, do not eat for at least 2 hours before taking PROCYSBI and at least 30 minutes after taking PROCYSBI.
• If you are not able to take PROCYSBI without eating, you can eat a small amount of food (½ cup) within 1 hour before you take PROCYSBI through 1 hour after you take it.
• Avoid eating foods that are high in fat close to the time that you will take a dose of PROCYSBI.
• Talk to your doctor or pharmacist if you have questions about how to take PROCYSBI.
• PROCYSBI is available as capsules in a bottle and oral granules in packets.

Capsules:

• Swallow PROCYSBI capsules whole. Do not crush or chew capsules or capsule contents.
• Take the PROCYSBI capsule whole with fruit juice (except for grapefruit juice) or water.
• If PROSCYBI capsules cannot be swallowed whole, the capsule may be opened and the contents sprinkled on and mixed in applesauce, berry jelly or fruit juice (except grapefruit juice) and taken by mouth.

Granules:

• Sprinkle and mix PROCYSBI oral granules on applesauce, berry jelly or fruit juice (except grapefruit juice) and take by mouth. Do not crush or chew oral granules.
• Read the "Instructions for Use" if you have a gastrostomy tube (G-tube).
• Take PROCYSBI at least 1 hour before or 1 hour after you take medicines that contain bicarbonate or carbonate.
• If you miss a dose, take it as soon as possible, up to 8 hours after the scheduled time of the missed dose. If it is less than 4 hours of the time the next dose is due, skip the missed dose. Take the next dose at your regularly scheduled time. Do not take 2 doses at one time to make up for a missed dose.
• If you take too much PROCYSBI, call your doctor or go to the nearest hospital emergency room right away.
• Your doctor should do blood tests to measure the amount of cystine inside your white blood cells or to measure the amount of PROCYSBI in your blood before you start taking it and regularly during treatment with PROCYSBI. This will help your doctor to prescribe the dose of PROCYSBI that is right for you.
• Take supplements if prescribed by your doctor, such as salt or mineral replacement supplements, vitamin D, or thyroid hormone.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Delayed-release capsules: 25 mg and 75 mg cysteamine
• Delayed-release oral granules: 75 mg and 300 mg cysteamine in single-use packets

This medicine is available in fallowing brand namesː

• PROSCYBI

What side effects can this medication cause?

The most common side effects of this medicine include:

• vomiting
• pink eye
• tiredness
• problems with body salts or electrolytes
• nausea
• diarrhea
• flu
• infection of ear, nose or throat
• stomach (abdominal) pain
• cold
• headache
• joint pain

PROCYSBI can cause serious side effects, including:

• Skin, bone, and joint problems
• Skin rash
• Stomach and bowel (intestinal) problems
• Central nervous system symptoms
• Low white blood cell count and certain abnormal liver function blood tests
• Benign intracranial hypertension

What special precautions should I follow?

• Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts. Reduce dosage if skin and bone lesions occur.
• Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue if severe skin rash such as erythema multiforme bullosa or toxic epidermal necrolysis occurs.
• Gastrointestinal (GI) ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
• Central Nervous System (CNS) symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
• Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell count and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal.
• Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema have been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

What to do in case of emergency/overdose?

Symptoms of overdosage may include:

• nausea and vomiting

Management of overdosage:

• The respiratory and cardiovascular systems should be supported appropriately.
• No specific antidote is known.
• Hemodialysis may be considered since cysteamine is poorly bound to plasma proteins.

Can this medicine be used in pregnancy?

• There are no available data on PROCYSBI use in pregnant women to inform any drug-associated risks for birth defects or miscarriage.

Can this medicine be used in children?

• The safety and effectiveness of PROCYSBI have been established in pediatric patients 1 year of age and older for the treatment of nephropathic cystinosis.
• The safety and effectiveness of PROCYSBI have not been established in patients less than 1 year of age.

What are the active and inactive ingredients in this medicine?

• Active ingredients: cysteamine bitartrate
• Inactive ingredients: Eudragit® L 30 D-55, hypromellose, microcrystalline cellulose, purified water, sodium lauryl sulfate, talc, and triethyl citrate. Capsule shell ingredients: gelatin, titanium dioxide, blue ink and white ink.

Who manufactures and distributes this medicine?

• Distributed by: Horizon Therapeutics USA, Inc., Deerfield, IL

What should I know about storage and disposal of this medication?

• Prior to dispensing, store PROCYSBI delayed-release capsules and PROCYSBI delayed-release oral granules in a refrigerator, 2°C to 8°C (36°F to 46°F).
• Dispense PROCYSBI delayed-release capules and PROCYSBI delayed-release oral granules with a 4 month discard date.
• Dispense in original packaging. Do not subdivide or repackage.
• Do not remove desiccant or oxygen absorber(s) from the bottle of PROCYSBI delayed-release capsules. Keep bottles tightly closed in a dry place.
• Protect from light and moisture.

• Store PROCYSBI delayed-release capsules and PROCYSBI delayed-relase oral granules at room temperature, 20°C to 25°C (68°F to 77°F) in the original packaging. Do not subdivide or repackage.
• Protect from light and moisture.
• Do not store PROCYSBI delayed-release oral granules in opened packets.

• Dailymed label info on Cysteamine bitartrate
• FDA Cysteamine bitartrate

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