Procysbi 300mg Oral Granules
It is used to treat nephropathic cystinosis.
Drug Class
Agent for metabolic disorders
Pharmacologic Class
Cystine-depleting agent; Aminothiol
Pregnancy Category
C
FDA Approved
May 2017
DEA Schedule
Not Controlled
Overview
What is this medicine?
Procysbi is a medicine used to treat a rare genetic disease called nephropathic cystinosis. In this disease, a substance called cystine builds up in different parts of the body, causing damage. Procysbi helps to remove this excess cystine from the body's cells, which can slow down or prevent damage to organs like the kidneys and eyes.
How to Use This Medicine
Taking Your Medication Correctly
To ensure you get the most benefit from your medication, follow these steps:
1. Follow Your Doctor's Instructions: Take this medication exactly as directed by your doctor. Read all the information provided with your medication and follow the instructions carefully.
2. Preparing Your Dose: Sprinkle the granules on up to 1/2 cup (4 ounces or 120 mL) of applesauce or berry jelly. Alternatively, you can mix the granules with 1/2 cup (4 ounces or 120 mL) of fruit juice, except for grapefruit juice. Do not crush or chew the granules.
3. Taking Your Dose: Take your dose within 30 minutes of mixing. It is recommended to take this medication at least 30 minutes before or 2 hours after a meal.
4. Food and Medication: 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 your dose. However, it is essential to be consistent and either always take your medication with a small amount of food or always take it on an empty stomach.
5. Avoiding High-Fat Foods: Try to avoid eating high-fat foods close to the time you take your medication.
6. Using a Feeding Tube: If you have a feeding tube, you can still use this medication. Follow the instructions provided by your healthcare provider and flush the feeding tube after administering the medication.
7. Taking Other Medications: If you take other medications, they may need to be taken at a different time than this medication. Consult with your doctor or pharmacist to determine the best schedule for taking your medications.
Storing and Disposing of Your Medication
1. Storage: Store your medication at room temperature, protected from light, and in a dry place. Do not store it in a bathroom.
2. Keeping it Safe: Keep your medication in its original packaging until you are ready to use it. Store all medications in a safe place, out of the reach of children and pets.
Missing a Dose
If you miss a dose, take it as soon as you remember. However, 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 ensure you get the most benefit from your medication, follow these steps:
1. Follow Your Doctor's Instructions: Take this medication exactly as directed by your doctor. Read all the information provided with your medication and follow the instructions carefully.
2. Preparing Your Dose: Sprinkle the granules on up to 1/2 cup (4 ounces or 120 mL) of applesauce or berry jelly. Alternatively, you can mix the granules with 1/2 cup (4 ounces or 120 mL) of fruit juice, except for grapefruit juice. Do not crush or chew the granules.
3. Taking Your Dose: Take your dose within 30 minutes of mixing. It is recommended to take this medication at least 30 minutes before or 2 hours after a meal.
4. Food and Medication: 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 your dose. However, it is essential to be consistent and either always take your medication with a small amount of food or always take it on an empty stomach.
5. Avoiding High-Fat Foods: Try to avoid eating high-fat foods close to the time you take your medication.
6. Using a Feeding Tube: If you have a feeding tube, you can still use this medication. Follow the instructions provided by your healthcare provider and flush the feeding tube after administering the medication.
7. Taking Other Medications: If you take other medications, they may need to be taken at a different time than this medication. Consult with your doctor or pharmacist to determine the best schedule for taking your medications.
Storing and Disposing of Your Medication
1. Storage: Store your medication at room temperature, protected from light, and in a dry place. Do not store it in a bathroom.
2. Keeping it Safe: Keep your medication in its original packaging until you are ready to use it. Store all medications in a safe place, out of the reach of children and pets.
Missing a Dose
If you miss a dose, take it as soon as you remember. However, 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 Procysbi exactly as prescribed by your doctor, usually twice a day, every 12 hours.
- Always take Procysbi with food to help reduce stomach upset.
- Do NOT chew or crush the granules. They should be sprinkled on a small amount of soft food (e.g., applesauce, yogurt, pureed fruit) and consumed immediately.
- Do not store the mixed food; discard any unused portion.
- Maintain regular appointments for blood tests (to check cystine levels, blood counts, and liver function) and eye exams.
- Stay well-hydrated as advised by your doctor.
- Report any unusual side effects, especially severe stomach upset, changes in vision, or signs of infection.
Available Forms & Alternatives
Dosing & Administration
Adult Dosing
Standard Dose:
Initial dose 1.3 g/m²/day (or 25 mg/kg/day) divided into two doses, administered every 12 hours. Dosing is adjusted based on leukocyte cystine levels.
Condition-Specific Dosing:
target_leukocyte_cystine:
<1 nmol ½ cystine/mg protein
Pediatric Dosing
Neonatal:
Not established (safety and efficacy not established in children less than 2 years of age for Procysbi granules, though cysteamine is used off-label in infants)
Infant:
Not established (safety and efficacy not established in children less than 2 years of age for Procysbi granules)
Child:
Initial dose 1.3 g/m²/day (or 25 mg/kg/day) divided into two doses, administered every 12 hours. Dosing is adjusted based on leukocyte cystine levels. Max dose 1.95 g/m²/day (or 50 mg/kg/day).
Adolescent:
Initial dose 1.3 g/m²/day (or 25 mg/kg/day) divided into two doses, administered every 12 hours. Dosing is adjusted based on leukocyte cystine levels. Max dose 1.95 g/m²/day (or 50 mg/kg/day).
Dose Adjustments
Renal Impairment:
Mild:
No specific dose adjustment recommended.
Moderate:
No specific dose adjustment recommended.
Severe:
Use with caution; monitor for adverse effects.
Dialysis:
Not available
Hepatic Impairment:
Mild:
No specific dose adjustment recommended.
Moderate:
No specific dose adjustment recommended.
Severe:
Use with caution; monitor for adverse effects. Consider dose reduction.
Note:
Patients with severe hepatic impairment may have increased systemic exposure to cysteamine.
Pharmacology
Mechanism of Action
Cysteamine is an aminothiol compound that reduces cystine accumulation in cells. It acts within the lysosome to convert cystine to cysteine and cysteine-cysteamine mixed disulfide. These products can then exit the lysosome via the amino acid transport system, thereby reducing intralysosomal cystine accumulation in patients with cystinosis.
Pharmacokinetics
Absorption:
Bioavailability:
Not available (relative bioavailability compared to IV not established)
Tmax:
Approximately 3-4 hours (granules)
FoodEffect:
High-fat meal delays Tmax and decreases Cmax, but AUC is similar. Administer with food to reduce gastrointestinal side effects.
Distribution:
Vd:
Approximately 1.5 L/kg
ProteinBinding:
Not extensively studied, but expected to be low due to its small molecular weight and hydrophilic nature.
CnssPenetration:
Limited (crosses the blood-brain barrier to some extent, but concentrations in CSF are lower than plasma)
Elimination:
HalfLife:
Approximately 4 hours
Clearance:
Not available
ExcretionRoute:
Primarily renal (as metabolites)
Unchanged:
<1% (renal)
Pharmacodynamics
OnsetOfAction:
Reduction in leukocyte cystine levels typically observed within weeks to months of initiating therapy.
PeakEffect:
Not directly applicable as it's a chronic treatment for cystine depletion.
DurationOfAction:
Requires continuous administration every 12 hours to maintain therapeutic effect.
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 medical attention immediately:
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 experience no side effects or only mild side effects while taking this medication. However, if you notice any of the following symptoms, contact your doctor or seek medical attention if they bother you or do not go away:
Diarrhea
Feeling tired or weak
Change in body odor
Bad breath
Eye irritation
Flu-like symptoms
Nose or throat irritation
* Ear pain
This is not an exhaustive list of possible side effects. 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 medical attention immediately:
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 experience no side effects or only mild side effects while taking this medication. However, if you notice any of the following symptoms, contact your doctor or seek medical attention if they bother you or do not go away:
Diarrhea
Feeling tired or weak
Change in body odor
Bad breath
Eye irritation
Flu-like symptoms
Nose or throat irritation
* Ear pain
This is not an exhaustive list of possible side effects. 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 diarrhea
- Unusual or severe abdominal pain
- Unexplained fever, chills, or sore throat (signs of infection)
- Unusual bleeding or bruising
- Yellowing of the skin or eyes (jaundice)
- Dark urine or pale stools
- Severe headache
- Skin rash or itching
- Changes in vision or eye discomfort
- Strong, unpleasant body odor or breath
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.
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 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, including starting, stopping, or adjusting the dose of any drug, consult with your doctor to confirm it is safe to do so.
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.
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 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, including starting, stopping, or adjusting the dose of any drug, consult with your doctor to confirm it is safe to do so.
Precautions & Cautions
Important Warnings and Cautions for Patients Taking This Medication
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. This will help ensure your safety and prevent potential interactions with other treatments.
Precautions and Monitoring
Avoid operating a vehicle or engaging in activities that require alertness until you understand how this medication affects you.
Regular blood tests will be necessary to monitor your condition, as directed by your doctor. Be sure to discuss any concerns or questions with your doctor.
Refrain from consuming alcohol while taking this medication, as it may increase the risk of adverse effects.
Potential Risks and Side Effects
This medication may increase the risk of stomach or bowel ulcers, as well as bleeding. Discuss this risk with your doctor and report any concerns.
Low white blood cell counts have been associated with this medication, which may increase the risk of infection. Seek immediate medical attention if you experience symptoms such as fever, chills, or sore throat.
A rare but severe skin reaction called toxic epidermal necrolysis may occur. Seek emergency medical help if you experience symptoms such as:
+ Red, swollen, blistered, or peeling skin (with or without fever)
+ Red or irritated eyes
+ Sores in your mouth, throat, nose, or eyes
Special Considerations
If you become pregnant or suspect you may be pregnant while taking this medication, contact your doctor immediately.
For pediatric patients, it is essential to monitor weight changes, as the dosage of this medication may need to be adjusted. Consult with your doctor if your child's weight changes.
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. This will help ensure your safety and prevent potential interactions with other treatments.
Precautions and Monitoring
Avoid operating a vehicle or engaging in activities that require alertness until you understand how this medication affects you.
Regular blood tests will be necessary to monitor your condition, as directed by your doctor. Be sure to discuss any concerns or questions with your doctor.
Refrain from consuming alcohol while taking this medication, as it may increase the risk of adverse effects.
Potential Risks and Side Effects
This medication may increase the risk of stomach or bowel ulcers, as well as bleeding. Discuss this risk with your doctor and report any concerns.
Low white blood cell counts have been associated with this medication, which may increase the risk of infection. Seek immediate medical attention if you experience symptoms such as fever, chills, or sore throat.
A rare but severe skin reaction called toxic epidermal necrolysis may occur. Seek emergency medical help if you experience symptoms such as:
+ Red, swollen, blistered, or peeling skin (with or without fever)
+ Red or irritated eyes
+ Sores in your mouth, throat, nose, or eyes
Special Considerations
If you become pregnant or suspect you may be pregnant while taking this medication, contact your doctor immediately.
For pediatric patients, it is essential to monitor weight changes, as the dosage of this medication may need to be adjusted. Consult with your doctor if your child's weight changes.
Overdose Information
Overdose Symptoms:
- Severe gastrointestinal upset (nausea, vomiting, diarrhea)
- Lethargy
- Hypotension
- Seizures
- Respiratory depression
- Liver dysfunction
- Bone marrow suppression
What to Do:
Seek immediate medical attention or call a poison control center. In the US, call 1-800-222-1222. Treatment is supportive and symptomatic.
Drug Interactions
Moderate Interactions
- Penicillamine (potential for additive toxicity, e.g., renal, hepatic, hematologic effects)
- Antacids (may affect absorption, separate administration)
Monitoring
Baseline Monitoring
Leukocyte cystine levels
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 (e.g., leukopenia, anemia) and establish baseline.
Timing: Prior to initiation of therapy
Liver Function Tests (LFTs) including ALT, AST, alkaline phosphatase, bilirubin
Rationale: To assess for potential hepatotoxicity and establish baseline.
Timing: Prior to initiation of therapy
Renal Function Tests (serum creatinine, BUN)
Rationale: To assess kidney function, especially important in cystinosis patients.
Timing: Prior to initiation of therapy
Ophthalmologic examination (slit lamp)
Rationale: To assess for corneal cystine crystals and other ocular manifestations of cystinosis.
Timing: Prior to initiation of therapy
Routine Monitoring
Leukocyte cystine levels
Frequency: Every 3 months initially, then every 6 months once stable
Target: <1 nmol ½ cystine/mg protein
Action Threshold: >1 nmol ½ cystine/mg protein (consider dose adjustment)
Complete Blood Count (CBC) with differential
Frequency: Every 3-6 months
Target: Within normal limits
Action Threshold: Significant decrease in WBC count (especially leukopenia), anemia, or thrombocytopenia (consider dose reduction or interruption)
Liver Function Tests (LFTs)
Frequency: Every 3-6 months
Target: Within normal limits
Action Threshold: Significant elevation of liver enzymes (consider dose reduction or interruption)
Renal Function Tests
Frequency: Every 6-12 months
Target: Monitor for progression of renal disease
Action Threshold: Significant decline in renal function
Ophthalmologic examination (slit lamp)
Frequency: Annually
Target: Monitor for corneal crystal accumulation
Action Threshold: Significant increase in crystal density or visual impairment
Symptom Monitoring
- Nausea
- Vomiting
- Diarrhea
- Abdominal pain
- Bad breath/body odor (sulfur-like)
- Fatigue
- Skin rash
- Headache
- Signs of infection (fever, sore throat) due to potential leukopenia
- Yellowing of skin/eyes (jaundice) or dark urine (signs of liver problems)
Special Patient Groups
Pregnancy
Category C. Animal studies have shown adverse effects on fetal development (skeletal abnormalities, cleft palate) at doses similar to or higher than human therapeutic doses. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus.
Trimester-Specific Risks:
First Trimester:
Potential for teratogenicity based on animal data.
Second Trimester:
Potential for developmental abnormalities.
Third Trimester:
Potential for developmental abnormalities.
Lactation
L3 (Moderately Safe). Cysteamine is excreted in the milk of lactating rats. It is unknown whether cysteamine is excreted in human milk. Due to the potential for serious adverse reactions in the breastfed infant, a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother.
Infant Risk:
Potential for adverse effects based on animal data and drug properties. Monitor breastfed infant for adverse effects.
Pediatric Use
Procysbi is indicated for the management of nephropathic cystinosis in adults and children 2 years of age and older. It is the primary population for this drug. Dosing is weight-based and adjusted according to leukocyte cystine levels. Safety and efficacy in children younger than 2 years of age have not been established for Procysbi granules.
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 granules must be taken every 12 hours, consistently, to maintain therapeutic cystine depletion.
- Always administer with food to minimize gastrointestinal side effects.
- Do not chew or crush the granules; they are designed for delayed release.
- Leukocyte cystine levels are the primary biomarker for dose adjustment and treatment efficacy.
- Patients may experience a sulfur-like body odor or bad breath, which is a common side effect of cysteamine.
- Regular ophthalmologic exams are crucial to monitor for corneal cystine crystal accumulation, even with systemic therapy.
Alternative Therapies
- Cysteamine bitartrate immediate-release capsules (Cystagon®)
- Cystaran® (cysteamine ophthalmic solution) for ocular manifestations
Cost & Coverage
Average Cost:
Extremely high (e.g., $30,000 - $50,000+) per 100 capsules/granules
Insurance Coverage:
Specialty Tier (requires prior authorization, often covered by major medical plans due to orphan drug status for a life-threatening condition)
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 facilitate prompt and effective treatment.