cysteamine (Rx)

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Brand and Other Names:Cystagon, Procysbi

Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

capsule, immediate-release (Cystagon)

  • 50mg
  • 150mg

capsule, delayed-release (Procysbi)

  • 25mg
  • 75mg
more...

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 (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/m⊃2/day if WBC cystine level remains higher than the target, or cysteamine concentration has not been achieved  
  • 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

Raise dose if leukocyte cystine level remains >2 nmol / ½ cystine/mg of protein

Dosing Considerations

Monitor leukocyte cystine levels

Because the measured WBC cystine concentration depends on the assays used for cystine and total protein content, individual patient sample concentration values from different assays may not be interchangeable

Procysbi: Obtain blood samples for WBC cystine concentration measurement 12 hr after dose

Cysteamine-naive patients: Obtain measurement after reaching the maintenance dose, then monthly for 3 months, quarterly for 1 year, and then twice-yearly, at a minimum

For patients switching from immediate-release cysteamine to Procysbi: Obtain measurement after 2 weeks of Procysbi treatment while titrating the dose, then quarterly for 6 months, then twice yearly, at a minimum

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
more...

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)

  • <2 years: Safety and efficacy not established
  • Cysteamine-naïve patients
    • Starting dose: 0.2-0.3 g/m²/day divided q12hr
    • 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
  • 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

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

Dosage Modifications

Raise dose if leukocyte cystine level remains >2 nmol / ½ cystine/mg of protein

Dosing Considerations

Monitor leukocyte cystine levels

Because the measured WBC cystine concentration depends on the assays used for cystine and total protein content, individual patient sample concentration values from different assays may not be interchangeable

Procysbi: Obtain blood samples for WBC cystine concentration measurement 12 hr after dose

Cysteamine-naive patients: Obtain measurement after reaching the maintenance dose, then monthly for 3 months, quarterly for 1 year, and then twice-yearly, at a minimum

For patients switching from immediate-release cysteamine to Procysbi: Obtain measurement after 2 weeks of Procysbi treatment while titrating the dose, then quarterly for 6 months, then twice yearly, at a minimum

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Adverse Effects

>10%

Vomiting (35%)

Anorexia (31%)

Diarrhea (16%)

Fever (11%)

Lethargy (11%)

1-10%

Rash (7%)

Frequency Not Defined

Seizures

Ataxia

Encephalophathy

Somnolence

Depression

Nightmare

Impaired cognition

Hypertension

Urticaria

Bad breath

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Warnings

Contraindications

Hypersensitivity to cysteamine or penicillamine

Cautions

Blood disorders, hepatic impairment, seizures

Withhold if mild/moderate rash develops and restart at lower dose with subsequent titration to therapeutic dose; do not rechallenge if severe skin rash (eg, erythema multiforme bullosa, toxic epidermal necrolysis) develops

Scoliosis, osteopenia, compression fractures accompanied by leg pain and joint hyperextension may occur

Do NOT give whole capsules to <6 years due to risk of aspiration, open capsule & sprinkle on food

Temporarily suspend if serious GI or CNS symptoms

Monitor: Leukocyte cystine levels

May cause abnormal liver function tests

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Pregnancy & Lactation

Pregnancy Category: C

Lactation: excretion in milk unknown/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.

more...
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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

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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

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, give with food and limit the amount of food to approximately 4 ounces within 1 hr before taking Procysbi through 1 hr after taking Procysbi; take Procysbi in a consistent manner in regard to food

Avoid high fat food close to dosing

Swallow whole with fruit juice (except grapefruit juice), do not crush or chew capsule or capsule contents

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
  • ≥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

Missed dose

  • If a dose is missed, take the dose as soon as possible up to 8 hr after the scheduled time
  • However, if a dose is missed and the next scheduled dose is due in <4 hr, do not take the missed dose and take the next dose at the usual scheduled time
  • Do not take 2 doses at the same time to make up for a missed dose
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Formulary

FormularyPatient Discounts

Adding plans allows you to compare formulary status to other drugs in the same class.

To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.

Adding plans allows you to:

  • View the formulary and any restrictions for each plan.
  • Manage and view all your plans together – even plans in different states.
  • Compare formulary status to other drugs in the same class.
  • Access your plan list on any device – mobile or desktop.

The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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