Dosing & Uses
Dosage Forms & Strengths
capsule, immediate-release (Cystagon)
- 50mg
- 150mg
capsule, delayed-release (Procysbi)
- 25mg
- 75mg
granules, delayed-release (Procysbi)
- 75mg/packet
- 300mg/packet
Nephropathic Cystinosis
Immediate-release capsule (Cystagon)
- Starting dose: One-sixth to one-fourth of target maintenance dose
- Maintenance: 2 g/day PO divided q6hr; dose should be reached after 4-6 weeks of incremental dosage increases; not to exceed 1.95 g/m²/day or 90 mg/kg/day
Delayed-release capsule or granules (Procysbi)
-
Cysteamine-naïve patients
- Starting dose: One-sixth to one-fourth of target maintenance dose
- Gradually increase dose over 4-6 weeks until maintenance dosage is achieved to help reduce the risk of adverse reactions
- Maintenance dose: 1.3 g/m2/day PO divided q12hr; may increase to 1.95 g/m2/day if WBC cystine level remains higher than the target, or cysteamine concentration has not been achieved
- If a patient experiences initial intolerance, temporarily discontinue and then restart at a lower dosage and gradually increase dosage
-
Dosage titration to therapeutic target WBC cystine concentration
- Measure WBC cystine concentration and titrate dose as needed to achieve the therapeutic target WBC cysteine concentration
- If a dose adjustment is required, increase dosage by 10%, rounded to nearest dosage that can be administered using available strengths of capsules or packets of oral granules
- If adverse reactions occur, decrease dosage; some patients may be unable to achieve their therapeutic target due to poor tolerability
-
Switching from immediate-release capsules to Procysbi
- Starting total daily dose of Procysbi should be equal to previous total daily dose of immediate-release cysteamine bitartrate
- Measure WBC cystine concentration 2 weeks after initiation
- Titrate dose as needed to achieve target WBC cystine concentrations
- Do not exceed 1.95 g/m2/day
Dosage Modifications
Renal impairment
- Increased exposure to cysteamine in patients with renal impairment compared to healthy subjects is not considered to be clinically meaningful
Dosing Considerations
Monitoring parameters
- Recommended target WBC cystine concentration: <1 nmol ½ cystine/mg protein (using mixed leukocyte assay)
- Cysteamine-naïve patients: Obtain WBC cystine concentration after reaching the maintenance Procysbi dose, then monthly for 3 months, quarterly for 1 year, and then twice-yearly, at a minimum
- Switching from immediate-release cysteamine to Procysbi: Obtain WBC cystine concentration 2 weeks after initiating treatment and continue monitoring if further dosage titration is required to achieve therapeutic target WBC cystine concentration
- Once the therapeutic target is achieved, continue monitoring quarterly for 6 months, then twice yearly, at a minimum
- Obtain WBC cystine concentration 12 hr after the patient’s last dose, before administration of the next dose (ie, trough concentration)
- It is important to accurately record the time of the last dose, the actual dose, and the time the blood sample was taken
Cystic Fibrosis (Orphan)
Lynovex: Orphan designation for cystic fibrosis
Sponsor
- NovaBiotics Ltd.; Cruickshank Building, Craibstone; Aberdeen AB21 9TR; United Kingdom
Huntington Disease (Orphan)
Orphan indication sponsor
- Bennu Pharmaceuticals, Inc (dual EMEA); 9 Commercial Blvd; Novato, CA 94949
Batten Disease (Orphan)
Treatment of neuronal ceroid lipofuscinoses (Batten disease)
Orphan indication sponsor
- Bennu Pharmaceuticals, Inc. (dual EMEA); 9 Commercial Blvd; Novato, CA 94949
Pancreatic Cancer (Orphan)
Orphan designation for treatment of pancreatic cancer
Orphan sponsor
- Raptor Pharmaceuticals, Inc.; 5 Hamilton Landing, Suite 160, Novato, CA 94949
Dosage Forms & Strengths
capsule, immediate-release (Cystagon)
- 50mg
- 150mg
capsule, delayed-release (Procysbi)
- 25mg
- 75mg
granules, delayed-release (Procysbi)
- 75mg/packet
- 300mg/packet
Nephropathic Cystinosis
Immediate-release capsule (Cystagon)
- Initial: give one-sixth to one-fourth of maintenace dose
- Maintenance (<12 years): 1.3 g/m²/day or 60 mg/kg/day of free base PO divided q6hr; not to exceed 1.95 g/m²/day or 90 mg/kg/day
- Maintenance (≥12 years or >110 lbs): 2 g/day PO divided q6hr; dose should be reached after 4-6 weeks of incremental dosage increases; not to exceed 1.95 g/m²/day or 90 mg/kg/day
Delayed-release capsule (Procysbi)
- <1 years: Safety and efficacy not established
- Initiate cysteamine treatment immediately after diagnosis of nephropathic cystinosis
-
Cysteamine-naïve patients
- Initial dose is a fraction of the maintenance dose (ie, one-sixth to one-fourth of the maintenance dose)
- Maintenance dose: 1.3 g/m²/day PO divided q12hr
- 1-6 years: Increase dose in 10% increments to the maintenance dosage, while monitoring WBC cystine concentrations; allow a minimum of 2 weeks between dosage adjustments; if patient achieves the therapeutic target WBC cystine concentration at a dosage below the recommended weight-based maintenance dosage, then stop dosage escalation and use the dosage as the patient's maintenance dosage
- ≥6 years: Gradually increase dose over 4-6 weeks until maintenance dose achieved
- If a patient experiences initial intolerance, temporarily discontinue drug and then restart at a lower dosage and gradually increase dosage
- After maintenance dosage achieved, the dose may need to be further increased to achieve a therapeutic target WBC cystine concentration; maximum dose is 1.95 g/m²/day
- Round dose calculations to the nearest incremental dosage that can be administered using the available capsule strengths; only use whole capsules
-
Switching from immediate-release capsules to Procysbi
- Starting total daily dose of Procysbi should be equal to previous total daily dose of immediate-release cysteamine bitartrate
- Measure WBC cystine concentration 2 weeks after initiation
- Titrate dose as needed to achieve target WBC cystine concentrations
Dosage Modifications
Renal impairment
- Increased exposure to cysteamine in patients with renal impairment compared to healthy subjects is not considered to be clinically meaningful
Dosing Considerations
Monitoring parameters
- Recommended target WBC cystine concentration: <1 nmol ½ cystine/mg protein (using mixed leukocyte assay)
- Cysteamine-naïve patients: Obtain WBC cystine concentration after reaching the maintenance Procysbi dose, then monthly for 3 months, quarterly for 1 year, and then twice-yearly, at a minimum
- Switching from immediate-release cysteamine to Procysbi: Obtain WBC cystine concentration 2 weeks after initiating treatment and continue monitoring if further dosage titration is required to achieve therapeutic target WBC cystine concentration
- Once the therapeutic target is achieved, continue monitoring quarterly for 6 months, then twice yearly, at a minimum
- Obtain WBC cystine concentration 12 hr after the patient’s last dose, before administration of the next dose (ie, trough concentration)
- It is important to accurately record the time of the last dose, the actual dose, and the time the blood sample was taken
Fanconi Syndrome (Off-label)
Delayed-release capsule (Procysbi)
Used to reduce cystine levels, potentially delaying kidney and other damage
Initial: Use one-sixth to one-fourth the maintenance dose
Maintenance: 1.3 g/m2/day PO divided q12hr; may increase to 1.95 g/m2/day if WBC cystine level remains higher than the target, or cysteamine concentration has not been achieved
Adverse Effects
>10%
Diarrhea
Nausea
Conjunctivus
Influenza
Gastroenteritis
Nasopharyngitis
Abdominal pain
Dehydration
Ear infection
Upper respiratory tract infection
Fatigue
Arthralgia
Cough
Pain in extremity
Immediate-release cysteamine
- Vomiting/emesis (12%)
Delayed-release cysteamine
- Vomiting/emesis (19-77%)
- Gastroenteritis/viral gastroenteritis (53%)
- Breath odor (24%)
- Nausea (16-18%)
- Abdominal pain/discomfort (14%)
- Electrolyte imbalance (12%)
- Headache (9-12%)
1-10%
Immediate-release cysteamine
- Nausea (7%)
- Anorexia/loss of appetite (5%)
Delayed-release cysteamine
- Dizziness (5%)
- Anorexia/loss of appetite (2%)
Postmarketing reports
Musculoskeletal: Joint hyperextension, leg pain, osteopenia, compression fracture, scoliosis, genu valgum
Skin: Erythema multiforme bullosa, toxic epidermal necrolysis, Ehlers-Danlos-like syndrome, molluscoid pseudotumors, skin striae, skin fragility
Central nervous system: seizures, lethargy, somnolence, depression and encephalopathy, benign intracranial hypertension (or PTC) and/or papilledema
Gastrointestinal: Fibrosing colonopathy
Warnings
Contraindications
Hypersensitivity to cysteamine or penicillamine
Cautions
Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts; these include molluscoid pseudotumors (purplish hemorrhagic lesions), skin striae, bone lesions (including osteopenia, compression fractures, scoliosis and genu valgum), leg pain, and joint hyperextension; monitor for development of skin or bone lesions and interrupt dosing if patients develop these lesions; restart at a lower dose under close supervision, then slowly increase to the appropriate therapeutic dose
Severe skin rashes (eg, erythema multiforme bullosa, toxic epidermal necrolysis) reported; if severe skin rashes develop, permanently discontinue use
Gastrointestinal (GI) ulceration and bleeding have been reported; GI tract symptoms including nausea, vomiting, anorexia and abdominal pain, sometimes severe; if severe GI tract symptoms develop, consider decreasing the Procysbi dose
May cause CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy; interrupt medication or adjust the dose as necessary for patients with severe symptoms or with symptoms that persist or progress; may impair their ability to perform tasks such as driving or operating machinery
Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels
Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema have been reported; monitor for signs and symptoms of PTC, including headache, tinnitus, dizziness, nausea, diplopia, blurry vision, loss of vision, pain behind the eye or pain with eye movement; if signs/symptoms persist, interrupt or decrease dose and refer to an ophthalmologist; if diagnosis is confirmed, permanently discontinue use
Fibrosing colonopathy
- Fibrosing colonopathy, including colonic stricture formation, reported in pediatric and young adult patients with nephropathic cystinosis; some of the patients had been treated for prolonged periods of time
- Reported symptoms include abdominal pain, vomiting, bloody or persistent diarrhea, and fecal incontinence; evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy; if diagnosis is confirmed, permanently discontinue therapy and switch to immediate-release cysteamine bitartrate capsules
- An association between methacrylic acid-ethyl acrylate copolymer (an inactive ingredient of this drug) and fibrosing colonopathy cannot be ruled out
Drug interaction overview
- Drugs that increase the gastric pH (eg, medications containing bicarbonate or carbonate) may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine and increase WBC cystine concentration; monitor WBC cystine concentration when drugs that increase the gastric pH are concomitantly used
- Consumption of alcohol with cysteamine may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and toxicities; therefore, do not consume alcoholic beverages during treatment
Pregnancy & Lactation
Pregnancy
There are no available data on use in pregnant women to inform any drug-associated risks for birth defects or miscarriage; use during pregnancy only if potential benefits justify potential risk to fetus
Cysteamine (administered as cysteamine bitartrate) was teratogenic and fetotoxic in rats at doses less than the recommended human maintenance dose
Lactation
There is no information on the presence of cysteamine in human milk, the effects on the breast-fed infant, or the effects on milk production
Cysteamine is present in the milk of lactating rats
Owing to the potential for serious adverse reactions in breastfed infants from cysteamine, breastfeeding is not recommended
Pregnancy Categories
A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.
B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk. C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done. D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk. X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist. NA: Information not available.Pharmacology
Mechanism of Action
Converts lysosomal cystine to cysteine & to cysteine-cysteamine mixed disulfide which then can exit lysosome in patients with cystinosis (inherited defect of lysosomal transport)
Pharmacokinetics
Onset: 1-1.8 hr
Duration: 6-12 hr
Peak plasma time: 1.4 hr
Vd: 156 L
Bioavailability: Rapidly absorbed
Protein Bound: 52% (mainly albumin)
Excretion: Small amount excreted in urine
Dialyzable: PD: yes, HD: no data
Administration
Oral Administration
Cystagon
- May open capsules and sprinkle on food
-
Missed dose
- If a dose is missed, take the dose as soon as possible; if it is within 2 hr of the next dose, skip the missed dose and go back to the regular dosing schedule
- Do not double the dose
Procysbi delayed-release capsules or granules
- Administer at least 1 hr before or after medications containing bicarbonate or carbonate
- Do not eat for at least 2 hr before taking Procysbi and for at least 30 minutes after to maximize absorption
- If patients are unable to take Procysbi without eating, take with food and limit the amount of food to ~4 ounces (1∕2 cup) within 1 hr before taking Procysbi through 1 hr after taking Procysbi
- Take in a consistent manner in regard to food
- Avoid high-fat food close to dosing
-
Unable to swallow capsule
- If unable to swallow, may open capsule and sprinkle intact granules on ~4 oz applesauce or berry jelly; eat mixture within 30 minutes, OR
- May disperse intact granules into small volume of either orange juice or apple juice (~4 oz), shake gently for 5 minutes, then administer by spoon or cup within 30 minutes
- Do not chew the granules
- ≥12 French G-tube: Mix intact granules with 4 oz applesauce and administer via feeding tube within 30 minutes; flush with ~8 oz or orange juice or apple juice to clear the tube
- Use mixtures within 30 min; do not save the mixtures for later use
-
Missed dose
- <8 hr after missed dose: Take dose as soon as possible
- <4 hr until next scheduled dose: Do not take missed dose; take next dose at the usual scheduled time
- Do not take 2 doses at the same time to make up for a missed dose
Storage
Immediate-release capsules
- Store at room temperature, 20-25ºC (68-77ºF) in the original packaging
Delayed-release capsules and granules
- Store at room temperature, 20-25ºC (68-77ºF) in the original packaging
- Do not subdivide or repackage
- Protect from light and moisture
- Do not store delayed-release oral granules in opened packets
Images
| BRAND | FORM. | UNIT PRICE | PILL IMAGE |
|---|---|---|---|
| Procysbi oral - | 75 mg capsule |  | |
| Procysbi oral - | 25 mg capsule |  |
Copyright © 2010 First DataBank, Inc.
Patient Handout
cysteamine bitartrate oral
CYSTEAMINE - ORAL
(sis-TAY-uh-meen)
COMMON BRAND NAME(S): Cystagon
USES: This medication is used to help preserve kidney function and manage kidney damage and other problems in people with an inherited disorder that causes build-up of a certain natural substance (cystine) in the body (nephropathic cystinosis). Cystine build-up can cause problems such as kidney problems, slow growth, weak bones, and eye problems. Cysteamine helps the body get rid of cystine.
HOW TO USE: Read the Patient Information Leaflet if available from your pharmacist before you start using cysteamine and each time you get a refill. If you have any questions, consult your doctor or pharmacist.Take this medication by mouth as directed by your doctor, usually 4 times a day.Take this medication with food or just after a meal to reduce stomach upset. Do not give the whole capsule to children younger than 6 years. Instead, open the capsule and sprinkle the contents onto soft food such as applesauce or mashed potatoes. Eat all of the mixture right away. Do not prepare a supply for future use.The dosage is based on weight, medical condition, and response to therapy. Your doctor may direct you to take a low dose at first, gradually increasing the dose to lower the chance of side effects such as upset stomach. Your doctor will adjust your dose based on your cystine levels to find the best dose for you. Follow your doctor's directions carefully.Take this medication regularly in order to get the most benefit from it. To help you remember, take it at the same times each day.
SIDE EFFECTS: Nausea, vomiting, fever, loss of appetite, tiredness, drowsiness, and diarrhea may occur. If any of these effects last or get worse, tell your doctor or pharmacist promptly.Remember that this medication has been prescribed because your doctor has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.Tell your doctor right away if you have any serious side effects, including: mental/mood changes (such as depression), deep sleep, seizures, unusual tiredness, headache, hearing problems (such as buzzing/ringing in the ears), dizziness, eye/vision problems (such as blurred vision, loss of vision, eye pain), skin problems (such as thinning of the skin, stretch marks, purple bleeding patches on the elbows), bone problems (such as bone/joint/back pain, leg pain, broken bones).Get medical help right away if you have any very serious side effects, including: black/bloody stools, stomach/abdominal pain, vomit that looks like coffee grounds.Cysteamine can commonly cause a rash that may not be serious. However, you may not be able to tell it apart from a rare rash that could be a sign of a severe reaction. Get medical help right away if you or your child develops any rash.A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.In the US -Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.
PRECAUTIONS: Before taking cysteamine, tell your doctor or pharmacist if you are allergic to it; or to penicillamine; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.Before using this medication, tell your doctor or pharmacist your or your child's medical history, especially of: liver disease, stomach/gut problems (such as bleeding, ulcers).This drug may make you dizzy or drowsy. Alcohol or marijuana (cannabis) can make you more dizzy or drowsy. Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages. Talk to your doctor if you are using marijuana (cannabis).During pregnancy, this medication should be used only when clearly needed. Discuss the risks and benefits with your doctor.It is unknown if this medication passes into breast milk. Consult your doctor before breast-feeding.
DRUG INTERACTIONS: Drug interactions may change how your medications work or increase your risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with your doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without your doctor's approval.
OVERDOSE: If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call their local poison control center at 1-800-222-1222. Canada residents can call a provincial poison control center.
NOTES: Do not share this medication with others.Lab and/or medical tests (such as cystine levels, complete blood count, electrolytes, kidney/liver function) should be done while you are taking this medication. Keep all medical and lab appointments. Consult your doctor for more details.
MISSED DOSE: If you miss a dose, take it as soon as you remember. If it is less than 2 hours before the next dose, skip the missed dose. Take your next dose at the regular time. Do not double the dose to catch up.
STORAGE: Store at room temperature away from light and moisture. Do not store in the bathroom. Keep all medications away from children and pets.Do not flush medications down the toilet or pour them into a drain unless instructed to do so. Properly discard this product when it is expired or no longer needed. Consult your pharmacist or local waste disposal company.
Information last revised December 2022. Copyright(c) 2023 First Databank, Inc.
IMPORTANT: HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.
Formulary
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