Procysbi 75mg Oral Granules
It is used to treat nephropathic cystinosis.
Drug Class
Cystine-depleting agent
Pharmacologic Class
Lysosomal cystine transport inhibitor
Pregnancy Category
Not available
FDA Approved
Apr 2013
DEA Schedule
Not Controlled
Overview
What is this medicine?
Procysbi is a medicine used to treat a rare genetic disease called nephropathic cystinosis. This disease causes a substance called cystine to build up in different parts of the body, leading to damage. Procysbi helps to reduce the amount of cystine in the body, which can slow down or prevent damage to organs like the kidneys and eyes.
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 medication and follow the instructions precisely.
Preparing and Taking Your Dose
To take your medication, 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 in 1/2 cup (4 ounces or 120 mL) of fruit juice, except for grapefruit juice. Do not crush or chew the granules. Take the dose within 30 minutes of mixing.
Timing and Food Considerations
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 your dose. It's essential to be consistent and either always take your medication with a small amount of food or always take it on an empty stomach. Avoid consuming high-fat foods close to the time you take your medication.
Special Instructions
If you have a feeding tube, you can use this medication as directed by your healthcare provider. After administering the medication through the feeding tube, be sure to flush the tube. If you take other medications, they may need to be taken at a different time than this medication. Consult your doctor or pharmacist to determine the best schedule for taking your medications.
Storing and Disposing of Your Medication
Store your medication at room temperature, protected from light, and in a dry place. Do not store it in a bathroom. Keep the 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.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if the next scheduled dose is less than 4 hours away, skip the missed dose and return to your regular dosing 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 medication and follow the instructions precisely.
Preparing and Taking Your Dose
To take your medication, 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 in 1/2 cup (4 ounces or 120 mL) of fruit juice, except for grapefruit juice. Do not crush or chew the granules. Take the dose within 30 minutes of mixing.
Timing and Food Considerations
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 your dose. It's essential to be consistent and either always take your medication with a small amount of food or always take it on an empty stomach. Avoid consuming high-fat foods close to the time you take your medication.
Special Instructions
If you have a feeding tube, you can use this medication as directed by your healthcare provider. After administering the medication through the feeding tube, be sure to flush the tube. If you take other medications, they may need to be taken at a different time than this medication. Consult your doctor or pharmacist to determine the best schedule for taking your medications.
Storing and Disposing of Your Medication
Store your medication at room temperature, protected from light, and in a dry place. Do not store it in a bathroom. Keep the 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.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if the next scheduled dose is less than 4 hours away, skip the missed dose and return to your regular dosing 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 every 12 hours.
- Always take Procysbi with food to help with absorption and reduce stomach upset.
- Do NOT chew, crush, or dissolve the granules. Swallow them whole or sprinkle them on a small amount of soft food (like applesauce or yogurt) and consume immediately.
- Avoid alcohol while taking Procysbi, as it can cause the medicine to be released too quickly.
- Maintain good hydration as recommended by your doctor.
- Be aware that this medication can cause a sulfur-like body odor or bad breath. Good hygiene may help.
Available Forms & Alternatives
Dosing & Administration
Adult Dosing
Standard Dose:
Initial dose 1.3 g/m²/day divided into two equal doses, administered every 12 hours. Titrate to maintain leukocyte cystine levels between 0.2 and 1.0 nmol ½ cystine/mg protein.
Condition-Specific Dosing:
Maximum Dose:
1.95 g/m²/day
Pediatric Dosing
Neonatal:
Not established
Infant:
Not established (approved for 2 years and older)
Child:
Initial dose 1.3 g/m²/day divided into two equal doses, administered every 12 hours. Titrate to maintain leukocyte cystine levels between 0.2 and 1.0 nmol ½ cystine/mg protein. For children 2 to less than 6 years of age, the recommended starting dose is 300 mg/day, increasing gradually. For children 6 years and older, the recommended starting dose is 600 mg/day, increasing gradually.
Adolescent:
Initial dose 1.3 g/m²/day divided into two equal doses, administered every 12 hours. Titrate to maintain leukocyte cystine levels between 0.2 and 1.0 nmol ½ cystine/mg protein.
Dose Adjustments
Renal Impairment:
Mild:
No specific dose adjustment recommended, use with caution.
Moderate:
No specific dose adjustment recommended, use with caution.
Severe:
No specific dose adjustment recommended, use with caution.
Dialysis:
Not available, use with caution.
Hepatic Impairment:
Mild:
No specific dose adjustment recommended, use with caution.
Moderate:
No specific dose adjustment recommended, use with caution.
Severe:
No specific dose adjustment recommended, use with caution.
Pharmacology
Mechanism of Action
Cysteamine is a cystine-depleting agent. It enters the lysosome and reacts with cystine to form cysteine and cysteine-cysteamine mixed disulfide. Both of these products can exit the lysosome via the intact transport system for cysteine, thereby reducing intralysosomal cystine accumulation in patients with cystinosis.
Pharmacokinetics
Absorption:
Bioavailability:
Not available (variable)
Tmax:
Approximately 4 hours (delayed-release)
FoodEffect:
Food increases absorption (AUC and Cmax) and reduces variability. Should be taken with food.
Distribution:
Vd:
Not available
ProteinBinding:
Not available
CnssPenetration:
Limited
Elimination:
HalfLife:
Approximately 4 hours
Clearance:
Not available
ExcretionRoute:
Renal (metabolites)
Unchanged:
Less than 1% (renal)
Pharmacodynamics
OnsetOfAction:
Not available (therapeutic effect is gradual, based on reduction of cystine accumulation)
PeakEffect:
Not available
DurationOfAction:
Not available (requires chronic 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 immediately or seek emergency 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 fainting
+ Increased thirst
+ Seizures
+ Feeling extremely tired or weak
+ Decreased appetite
+ Unable to pass urine or changes in urine output
+ Dry mouth
+ Dry eyes
+ Severe stomach upset or vomiting
Ringing in the ears
Dizziness or fainting
Feeling excessively 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 fractures or deformities
Fibrosing colonopathy, a severe bowel condition, which may cause:
+ Stomach pain
+ Vomiting
+ Weight loss
+ Bloody or prolonged diarrhea
+ Loss of bowel control
Other Possible Side Effects
Like all medications, this drug can cause side effects. While many people may not experience any side effects or only minor ones, it's essential to discuss any concerns with your doctor. If you experience any of the following side effects, contact your doctor if they bother you or do not go away:
Diarrhea
Feeling tired or weak
Changes 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, 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 immediately or seek emergency 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 fainting
+ Increased thirst
+ Seizures
+ Feeling extremely tired or weak
+ Decreased appetite
+ Unable to pass urine or changes in urine output
+ Dry mouth
+ Dry eyes
+ Severe stomach upset or vomiting
Ringing in the ears
Dizziness or fainting
Feeling excessively 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 fractures or deformities
Fibrosing colonopathy, a severe bowel condition, which may cause:
+ Stomach pain
+ Vomiting
+ Weight loss
+ Bloody or prolonged diarrhea
+ Loss of bowel control
Other Possible Side Effects
Like all medications, this drug can cause side effects. While many people may not experience any side effects or only minor ones, it's essential to discuss any concerns with your doctor. If you experience any of the following side effects, contact your doctor if they bother you or do not go away:
Diarrhea
Feeling tired or weak
Changes 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, 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
- Unusual tiredness or weakness
- Yellowing of the skin or eyes (jaundice)
- Dark urine
- Easy bruising or bleeding
- New or worsening skin rash or lesions
- Bone pain or joint pain
- Changes in vision
- Seizures
- Confusion or changes in behavior
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. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
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 health problems you have, 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 it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dose of any medication
Your doctor will help you determine if 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. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
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 health problems you have, 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 it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dose of any medication
Your doctor will help you determine if it is safe to take this medication with your other medications and health conditions.
Precautions & Cautions
Important Information for Patients Taking This Medication
It is crucial that you inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
Until you understand how this medication affects you, avoid operating a vehicle or engaging in any activities that require your full attention.
Regular blood tests will be necessary as directed by your doctor. Be sure to discuss any concerns or questions you have with your doctor during these appointments.
To minimize potential interactions, refrain from consuming alcohol while taking this medication.
Be aware that this medication may increase your risk of developing stomach or bowel ulcers, or bleeding. Discuss any concerns with your doctor.
There is a risk of developing low white blood cell counts, which can increase your susceptibility to infections. If you experience symptoms such as fever, chills, or a sore throat, contact your doctor immediately.
A rare but severe skin reaction known as toxic epidermal necrolysis can occur. This condition can lead to serious health issues, including death. Seek immediate medical attention if you notice any of the following symptoms: 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 are pregnant or become pregnant while taking this medication, notify your doctor right away.
Special Considerations for Children
If your child experiences any weight changes while taking this medication, consult with their doctor. The dosage may need to be adjusted accordingly.
It is crucial that you inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
Until you understand how this medication affects you, avoid operating a vehicle or engaging in any activities that require your full attention.
Regular blood tests will be necessary as directed by your doctor. Be sure to discuss any concerns or questions you have with your doctor during these appointments.
To minimize potential interactions, refrain from consuming alcohol while taking this medication.
Be aware that this medication may increase your risk of developing stomach or bowel ulcers, or bleeding. Discuss any concerns with your doctor.
There is a risk of developing low white blood cell counts, which can increase your susceptibility to infections. If you experience symptoms such as fever, chills, or a sore throat, contact your doctor immediately.
A rare but severe skin reaction known as toxic epidermal necrolysis can occur. This condition can lead to serious health issues, including death. Seek immediate medical attention if you notice any of the following symptoms: 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 are pregnant or become pregnant while taking this medication, notify your doctor right away.
Special Considerations for Children
If your child experiences any weight changes while taking this medication, consult with their doctor. The dosage may need to be adjusted accordingly.
Overdose Information
Overdose Symptoms:
- Severe gastrointestinal upset (nausea, vomiting, diarrhea)
- Lethargy
- Hypotension
- Seizures
- Respiratory depression
What to Do:
Seek immediate medical attention or call a poison control center. Call 1-800-222-1222
Drug Interactions
Moderate Interactions
- Proton pump inhibitors (PPIs): May cause premature release of cysteamine from the delayed-release granules, potentially leading to increased systemic exposure and adverse effects. Avoid concomitant use if possible. If co-administration is necessary, monitor for adverse effects.
- Alcohol: May cause premature release of cysteamine from the delayed-release granules. Avoid alcohol consumption.
Monitoring
Baseline Monitoring
Leukocyte cystine level
Rationale: To establish baseline and guide initial dosing.
Timing: Prior to initiation of therapy
Liver function tests (ALT, AST, bilirubin)
Rationale: To assess baseline hepatic function.
Timing: Prior to initiation of therapy
Complete Blood Count (CBC) with differential
Rationale: To assess baseline hematologic status.
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 3-6 months (or as needed for dose adjustments)
Target: 0.2 to 1.0 nmol ½ cystine/mg protein
Action Threshold: Levels outside target range require dose adjustment.
Liver function tests (ALT, AST, bilirubin)
Frequency: Every 6 months (or as clinically indicated)
Target: Within normal limits
Action Threshold: Significant elevations may require dose reduction or discontinuation.
Complete Blood Count (CBC) with differential
Frequency: Every 6 months (or as clinically indicated)
Target: Within normal limits
Action Threshold: Significant abnormalities (e.g., leukopenia, anemia) may require dose reduction or discontinuation.
Ophthalmologic examination (slit lamp)
Frequency: Annually
Target: Not applicable
Action Threshold: Progression of corneal crystals may indicate inadequate control.
Neurological assessment
Frequency: Periodically (as clinically indicated)
Target: Not applicable
Action Threshold: New or worsening neurological symptoms (e.g., seizures, lethargy, encephalopathy) require investigation.
Symptom Monitoring
- Nausea
- Vomiting
- Diarrhea
- Abdominal pain
- Bad breath/body odor
- Lethargy
- Headache
- Rash
- Skin lesions
- Bone pain
- Joint pain
- Visual changes
- Seizures
- Changes in mental status
Special Patient Groups
Pregnancy
Cysteamine may cause fetal harm when administered to a pregnant woman. Animal studies have shown teratogenic effects. 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 adverse developmental effects.
Third Trimester:
Potential for adverse developmental effects.
Lactation
It is not known whether cysteamine is excreted in human milk. Because of the potential for serious adverse reactions in breastfed infants, breastfeeding is not recommended during treatment with Procysbi.
Infant Risk:
Unknown, but potential for serious adverse reactions.
Pediatric Use
Procysbi is approved for patients 2 years of age and older. Dosing is weight-based and requires careful titration based on leukocyte cystine levels. Safety and effectiveness in children younger than 2 years have not been established.
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 is a delayed-release formulation of cysteamine bitartrate, designed to allow for twice-daily dosing compared to the immediate-release formulation (Cystagon) which requires four-times-daily dosing.
- Adherence to the prescribed dosing regimen is critical for effective management of cystinosis and prevention of long-term complications.
- Regular monitoring of leukocyte cystine levels is essential to ensure therapeutic efficacy and guide dose adjustments.
- Patients and caregivers should be educated on the proper administration technique (not chewing/crushing granules) and the importance of taking the medication with food.
- The characteristic sulfur-like odor associated with cysteamine can be a challenge for patients and may impact adherence. Discuss strategies for managing this with patients.
Alternative Therapies
- Cysteamine bitartrate immediate-release (Cystagon)
- Kidney transplantation (for end-stage renal disease due to cystinosis)
Cost & Coverage
Average Cost:
Very high per bottle
Insurance Coverage:
Specialty Tier (requires prior authorization)
General Drug Facts
If your symptoms or health issues persist or worsen, it is essential to contact your doctor promptly. To ensure safe and effective treatment, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so it is a good idea to consult with your pharmacist. If you have any questions or concerns about this medication, do not 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 reporting the incident, be prepared to provide detailed information, including the name of the medication taken, the amount, and the time it occurred.