Procysbi 75mg Capsules
It is used to treat nephropathic cystinosis.
Drug Class
Cystine-depleting agent
Pharmacologic Class
Aminothiol
Pregnancy Category
Category C
FDA Approved
Aug 2013
DEA Schedule
Not Controlled
Overview
What is this medicine?
Procysbi is a medicine used to treat a rare genetic condition called nephropathic cystinosis. In this condition, a substance called cystine builds up in different parts of the body, causing damage, especially to the kidneys and eyes. Procysbi helps to remove this harmful cystine from the body's cells, slowing down the damage and improving health.
How to Use This Medicine
Taking Your Medication Correctly
To get the most benefit from your medication, follow your doctor's instructions carefully. Read all the information provided with your prescription and follow the instructions closely.
Take your medication as directed by your doctor. Swallow the capsule whole - do not chew or crush it.
You can take your medication with fruit juice (except grapefruit juice) or water.
If you have trouble swallowing the capsule, you can sprinkle the contents on up to 1/2 cup (4 ounces/120 mL) of applesauce or berry jelly. Alternatively, you can mix the contents with 1/2 cup (4 ounces/120 mL) of fruit juice (except grapefruit juice). Do not chew the mixture. Take it within 30 minutes of preparing it.
Timing and Food
Take your medication at least 30 minutes before or 2 hours after a meal.
If you need to eat before taking your medication, you can have a small amount of food (about 1/2 cup) within 1 hour before or after taking it.
Always take your medication with a small amount of food or always take it on an empty stomach. Be consistent in your approach.
Avoid eating high-fat foods close to the time you take your medication.
Special Instructions
If you have a feeding tube, you can still take this medication. Follow the instructions provided by your healthcare provider. Flush the feeding tube after administering the medication.
If you take other medications, they may need to be taken at a different time than your current medication. Check with your doctor or pharmacist to determine the best schedule.
Storing and Disposing of Your Medication
Store your medication at room temperature, protected from light and moisture.
Keep the medication in its original container, with the lid tightly closed. Do not remove the antimoisture cube or packet.
Store the medication in a dry place, away from the bathroom.
Missing a Dose
If you miss a dose, take it as soon as you remember.
If the next dose is less than 4 hours away, skip the missed dose and return to your regular schedule.
* Do not take two doses at the same time or take extra doses.
To get the most benefit from your medication, follow your doctor's instructions carefully. Read all the information provided with your prescription and follow the instructions closely.
Take your medication as directed by your doctor. Swallow the capsule whole - do not chew or crush it.
You can take your medication with fruit juice (except grapefruit juice) or water.
If you have trouble swallowing the capsule, you can sprinkle the contents on up to 1/2 cup (4 ounces/120 mL) of applesauce or berry jelly. Alternatively, you can mix the contents with 1/2 cup (4 ounces/120 mL) of fruit juice (except grapefruit juice). Do not chew the mixture. Take it within 30 minutes of preparing it.
Timing and Food
Take your medication at least 30 minutes before or 2 hours after a meal.
If you need to eat before taking your medication, you can have a small amount of food (about 1/2 cup) within 1 hour before or after taking it.
Always take your medication with a small amount of food or always take it on an empty stomach. Be consistent in your approach.
Avoid eating high-fat foods close to the time you take your medication.
Special Instructions
If you have a feeding tube, you can still take this medication. Follow the instructions provided by your healthcare provider. Flush the feeding tube after administering the medication.
If you take other medications, they may need to be taken at a different time than your current medication. Check with your doctor or pharmacist to determine the best schedule.
Storing and Disposing of Your Medication
Store your medication at room temperature, protected from light and moisture.
Keep the medication in its original container, with the lid tightly closed. Do not remove the antimoisture cube or packet.
Store the medication in a dry place, away from the bathroom.
Missing a Dose
If you miss a dose, take it as soon as you remember.
If the next dose is less than 4 hours away, skip the missed dose and return to your regular schedule.
* Do not take two doses at the same time or take extra doses.
Lifestyle & Tips
- Take capsules whole; do not crush, chew, or open them. This is important because the capsule is designed to release the medicine slowly and protect it from stomach acid.
- Take with food to help reduce stomach upset.
- Take consistently as prescribed, usually twice a day, to maintain stable levels of the medicine in your body.
- Do not take antacids within one hour before or after taking Procysbi, as they can interfere with how the medicine works.
- Attend all scheduled doctor appointments and laboratory tests to monitor your condition and the effectiveness of the treatment.
Available Forms & Alternatives
Dosing & Administration
Adult Dosing
Standard Dose:
Initial dose 6 mg/kg/day divided into 2 doses. Titrate over 4-6 weeks to a target dose of 1.3 g/m²/day (approximately 20 mg/kg/day) divided into 2 doses. Max dose 1.95 g/m²/day or 2 g/day.
Condition-Specific Dosing:
targetLeukocyteCystine:
Maintain leukocyte cystine levels between 0.5 and 1.0 nmol ½ cystine/mg protein.
Pediatric Dosing
Neonatal:
Not established (approved for children 2 years and older)
Infant:
Not established (approved for children 2 years and older)
Child:
Initial dose 6 mg/kg/day divided into 2 doses. Titrate over 4-6 weeks to a target dose of 1.3 g/m²/day (approximately 20 mg/kg/day) divided into 2 doses. Max dose 1.95 g/m²/day or 2 g/day. For children < 10 kg, consider using immediate-release cysteamine.
Adolescent:
Initial dose 6 mg/kg/day divided into 2 doses. Titrate over 4-6 weeks to a target dose of 1.3 g/m²/day (approximately 20 mg/kg/day) divided into 2 doses. Max dose 1.95 g/m²/day or 2 g/day.
Dose Adjustments
Renal Impairment:
Mild:
No specific dose adjustment recommended, monitor closely.
Moderate:
No specific dose adjustment recommended, monitor closely.
Severe:
No specific dose adjustment recommended, monitor closely.
Dialysis:
Not available. Cysteamine is dialyzable, but specific recommendations for dosing in dialysis patients are not provided. Monitor leukocyte cystine levels closely.
Hepatic Impairment:
Mild:
No specific dose adjustment recommended, monitor closely.
Moderate:
No specific dose adjustment recommended, monitor closely.
Severe:
No specific dose adjustment recommended, monitor closely.
Pharmacology
Mechanism of Action
Cysteamine is an aminothiol that reduces cystine accumulation in cells. It enters lysosomes and reacts with cystine to form cysteine and cysteine-cysteamine mixed disulfide. These products can then exit the lysosome via specific transport systems, thereby depleting intralysosomal cystine stores.
Pharmacokinetics
Absorption:
Bioavailability:
Not directly quantified as a percentage, but well absorbed.
Tmax:
Approximately 3-4 hours (delayed-release formulation)
FoodEffect:
Food increases Tmax and AUC, but does not significantly affect Cmax. Administer with food to reduce gastrointestinal side effects.
Distribution:
Vd:
Not widely reported
ProteinBinding:
Limited
CnssPenetration:
Limited, but some penetration occurs.
Elimination:
HalfLife:
Approximately 4 hours
Clearance:
Not widely reported
ExcretionRoute:
Renal (as metabolites)
Unchanged:
Minimal
Pharmacodynamics
OnsetOfAction:
Weeks to months (to achieve stable leukocyte cystine levels)
PeakEffect:
Not applicable for chronic disease management; effect is sustained with consistent dosing.
DurationOfAction:
Not applicable; requires continuous administration.
Safety & Warnings
Side Effects
Urgent Side Effects: Seek Medical Help Right Away
Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor or seek immediate medical attention:
Signs of an allergic reaction, such as:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin with or without fever
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Signs of fluid and electrolyte problems, including:
+ Mood changes
+ Confusion
+ Muscle pain or weakness
+ Fast or abnormal heartbeat
+ Severe dizziness or passing out
+ Increased thirst
+ Seizures
+ Feeling very tired or weak
+ Decreased appetite
+ Unable to pass urine or changes in urine production
+ Dry mouth
+ Dry eyes
+ Severe upset stomach or vomiting
Ringing in the ears
Dizziness or passing out
Feeling sleepy
Eye pain
Stomach pain, black, tarry, or bloody stools, vomiting blood, or coffee ground-like vomit
Upset stomach or vomiting
Decreased appetite
Depression
Raised pressure in the brain, which may cause:
+ Headache
+ Blurred vision
+ Double vision
+ Loss of vision (in some cases, this may be permanent)
Severe skin problems, including:
+ Stretch marks
+ Joint problems
+ Bone problems, such as broken or deformed bones
Fibrosing colonopathy, a severe bowel problem, which may cause:
+ Stomach pain
+ Vomiting
+ Weight loss
+ Bloody or long-lasting diarrhea
+ Loss of bowel control
Other Possible Side Effects
Most people do not experience severe side effects, and some may only have minor side effects. However, if you notice any of the following symptoms or if they persist or bother you, contact your doctor or seek medical attention:
Diarrhea
Feeling tired or weak
Change in body odor
Bad breath
Eye irritation
Flu-like symptoms
Nose or throat irritation
* Ear pain
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects not mentioned here. If you have questions or concerns about side effects, contact your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor or seek immediate medical attention:
Signs of an allergic reaction, such as:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin with or without fever
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Signs of fluid and electrolyte problems, including:
+ Mood changes
+ Confusion
+ Muscle pain or weakness
+ Fast or abnormal heartbeat
+ Severe dizziness or passing out
+ Increased thirst
+ Seizures
+ Feeling very tired or weak
+ Decreased appetite
+ Unable to pass urine or changes in urine production
+ Dry mouth
+ Dry eyes
+ Severe upset stomach or vomiting
Ringing in the ears
Dizziness or passing out
Feeling sleepy
Eye pain
Stomach pain, black, tarry, or bloody stools, vomiting blood, or coffee ground-like vomit
Upset stomach or vomiting
Decreased appetite
Depression
Raised pressure in the brain, which may cause:
+ Headache
+ Blurred vision
+ Double vision
+ Loss of vision (in some cases, this may be permanent)
Severe skin problems, including:
+ Stretch marks
+ Joint problems
+ Bone problems, such as broken or deformed bones
Fibrosing colonopathy, a severe bowel problem, which may cause:
+ Stomach pain
+ Vomiting
+ Weight loss
+ Bloody or long-lasting diarrhea
+ Loss of bowel control
Other Possible Side Effects
Most people do not experience severe side effects, and some may only have minor side effects. However, if you notice any of the following symptoms or if they persist or bother you, contact your doctor or seek medical attention:
Diarrhea
Feeling tired or weak
Change in body odor
Bad breath
Eye irritation
Flu-like symptoms
Nose or throat irritation
* Ear pain
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects not mentioned here. If you have questions or concerns about side effects, contact your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Seek Immediate Medical Attention If You Experience:
- Severe nausea, vomiting, or abdominal pain
- Diarrhea that is severe or persistent
- Skin rash, lesions, or unusual skin changes
- Extreme tiredness, lethargy, or confusion
- Seizures or tremors
- Unexplained fever, sore throat, or signs of infection
- Unusual bleeding or bruising
- Severe headache
Before Using This Medicine
Before Taking This Medication: Important Information to Share with Your Doctor
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
If you are breastfeeding, as you should not breastfeed while taking this medication.
To ensure safe treatment, it is crucial to discuss the following with your doctor and pharmacist:
All medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins.
Any existing health problems, as this medication may interact with other drugs or health conditions.
Before making any changes to your medication regimen, consult with your doctor to confirm that it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dosage of any medication
Your doctor will help you determine whether it is safe to take this medication with your other medications and health conditions.
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
If you are breastfeeding, as you should not breastfeed while taking this medication.
To ensure safe treatment, it is crucial to discuss the following with your doctor and pharmacist:
All medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins.
Any existing health problems, as this medication may interact with other drugs or health conditions.
Before making any changes to your medication regimen, consult with your doctor to confirm that it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dosage of any medication
Your doctor will help you determine whether it is safe to take this medication with your other medications and health conditions.
Precautions & Cautions
Important Warnings and Cautions for Patients Taking This Medication
If you are taking this medication, it is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, about your treatment.
To ensure your safety, avoid driving and engaging in activities that require alertness until you understand how this medication affects you.
Regular blood tests will be necessary, as directed by your doctor. Be sure to discuss any concerns or questions you have with your doctor.
It is recommended that you avoid consuming alcohol while taking this medication, as it may increase the risk of adverse effects.
Be aware that this medication may increase the risk of stomach or bowel ulcers, as well as bleeding. Discuss any concerns with your doctor.
Additionally, this medication can cause a decrease in white blood cell count, which may increase your risk of infection. If you experience symptoms such as fever, chills, or sore throat, contact your doctor immediately.
In rare cases, a severe skin reaction called toxic epidermal necrolysis can occur, which can lead to serious health problems or even death. Seek medical attention immediately if you experience symptoms such as red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
If you become pregnant or suspect you are pregnant while taking this medication, contact your doctor right away.
Special Considerations for Children
If your child is taking this medication and experiences any changes in weight, consult with your doctor, as the dosage may need to be adjusted.
If you are taking this medication, it is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, about your treatment.
To ensure your safety, avoid driving and engaging in activities that require alertness until you understand how this medication affects you.
Regular blood tests will be necessary, as directed by your doctor. Be sure to discuss any concerns or questions you have with your doctor.
It is recommended that you avoid consuming alcohol while taking this medication, as it may increase the risk of adverse effects.
Be aware that this medication may increase the risk of stomach or bowel ulcers, as well as bleeding. Discuss any concerns with your doctor.
Additionally, this medication can cause a decrease in white blood cell count, which may increase your risk of infection. If you experience symptoms such as fever, chills, or sore throat, contact your doctor immediately.
In rare cases, a severe skin reaction called toxic epidermal necrolysis can occur, which can lead to serious health problems or even death. Seek medical attention immediately if you experience symptoms such as red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
If you become pregnant or suspect you are pregnant while taking this medication, contact your doctor right away.
Special Considerations for Children
If your child is taking this medication and experiences any changes in weight, consult with your doctor, as the dosage may need to be adjusted.
Overdose Information
Overdose Symptoms:
- Severe gastrointestinal upset (nausea, vomiting, abdominal pain)
- Lethargy, somnolence
- Seizures
- Bone marrow suppression (e.g., leukopenia, thrombocytopenia)
- Liver enzyme elevations
What to Do:
Seek immediate medical attention. Call 911 or your local emergency number. For poison control, call 1-800-222-1222.
Drug Interactions
Moderate Interactions
- Antacids (may prematurely dissolve the enteric coating of delayed-release capsules, leading to increased systemic absorption and potential for increased adverse effects)
- Drugs that cause gastrointestinal irritation (e.g., NSAIDs, corticosteroids) due to increased risk of GI adverse events when co-administered with cysteamine.
Monitoring
Baseline Monitoring
Leukocyte cystine level
Rationale: To establish baseline and guide initial dosing.
Timing: Prior to initiation of therapy.
Complete Blood Count (CBC) with differential
Rationale: To assess for potential bone marrow suppression.
Timing: Prior to initiation of therapy.
Liver Function Tests (LFTs)
Rationale: To assess hepatic function.
Timing: Prior to initiation of therapy.
Renal Function Tests (e.g., serum creatinine, BUN)
Rationale: To assess renal function.
Timing: Prior to initiation of therapy.
Ophthalmologic examination (slit lamp)
Rationale: To assess for corneal cystine crystals.
Timing: Prior to initiation of therapy.
Routine Monitoring
Leukocyte cystine level
Frequency: Every 3 months until stable, then every 6-12 months.
Target: 0.5 to 1.0 nmol ½ cystine/mg protein
Action Threshold: Adjust dose if levels are outside target range.
Complete Blood Count (CBC) with differential
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated).
Target: Within normal limits
Action Threshold: Investigate and manage significant abnormalities (e.g., leukopenia, anemia).
Liver Function Tests (LFTs)
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated).
Target: Within normal limits
Action Threshold: Investigate and manage significant abnormalities.
Renal Function Tests
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated).
Target: Within normal limits
Action Threshold: Investigate and manage significant abnormalities.
Ophthalmologic examination (slit lamp)
Frequency: Annually.
Target: Not applicable
Action Threshold: Monitor for progression or regression of corneal crystals.
Symptom Monitoring
- Gastrointestinal symptoms (nausea, vomiting, abdominal pain, diarrhea)
- Skin rash or lesions
- Central nervous system symptoms (lethargy, somnolence, seizures, headache, tremor)
- Musculoskeletal pain (e.g., leg pain)
- Fever, sore throat, or other signs of infection (due to potential bone marrow suppression)
Special Patient Groups
Pregnancy
Cysteamine is Pregnancy Category C. Animal studies have shown adverse effects on the fetus. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus. The underlying disease (cystinosis) can also have adverse effects on pregnancy outcomes.
Trimester-Specific Risks:
First Trimester:
Potential for teratogenicity based on animal data.
Second Trimester:
Potential for adverse developmental effects.
Third Trimester:
Potential for adverse developmental effects.
Lactation
Cysteamine is excreted into human milk. The decision to breastfeed during treatment should consider the importance of the drug to the mother, the potential for adverse effects on the breastfed infant, and the mother's underlying clinical condition. Monitor breastfed infants for potential adverse effects (e.g., gastrointestinal upset, rash).
Infant Risk:
L3 (Moderately Safe) - Monitor infant for adverse effects.
Pediatric Use
Procysbi is approved for children 2 years of age and older. Dosing is weight-based and requires careful titration. For children under 10 kg, immediate-release cysteamine (Cystagon) may be preferred due to dosing flexibility.
Geriatric Use
Clinical studies of Procysbi did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.
Clinical Information
Clinical Pearls
- Procysbi capsules must be swallowed whole; they should not be crushed, chewed, or opened, as this will destroy the enteric coating and alter drug release.
- Always administer Procysbi with food to minimize gastrointestinal side effects.
- Strict adherence to the dosing schedule is crucial for maintaining stable leukocyte cystine levels and optimizing therapeutic outcomes.
- Regular monitoring of leukocyte cystine levels is essential to guide dose adjustments and ensure therapeutic efficacy.
- Patients should be educated on the importance of avoiding antacids within one hour of taking Procysbi.
- This is a lifelong therapy for nephropathic cystinosis; patient and caregiver education on chronic management is vital.
Alternative Therapies
- Cysteamine bitartrate immediate-release capsules (e.g., Cystagon, generics)
Cost & Coverage
Average Cost:
Highly variable, typically several thousand USD per 100 capsules
Insurance Coverage:
Specialty Tier (Tier 4 or higher), often requires prior authorization and may have high co-pays/co-insurance.
General Drug Facts
If your symptoms or health issues persist or worsen, it is essential to contact your doctor for further guidance. To ensure your safety, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so it's a good idea to check with your pharmacist for more information. If you have any questions or concerns about this medication, don't hesitate to discuss them with your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek emergency medical attention. When seeking help, be prepared to provide details about the medication, including the amount taken and the time it happened, to help healthcare professionals provide the best possible care.