Cholbam 50mg Capsules
It is used to treat certain bile acid problems caused by single enzyme defects (SEDs).It is used to treat peroxisomal disorders like Zellweger spectrum disorders.
Drug Class
Bile acid replacement therapy
Pharmacologic Class
Bile acid
Pregnancy Category
Not available
FDA Approved
Mar 2015
DEA Schedule
Not Controlled
Overview
What is this medicine?
Cholbam is a medicine that contains a natural substance called cholic acid, which is a type of bile acid. It is used to treat rare genetic conditions where your body cannot make enough of its own bile acids. This medicine helps your body make and use bile acids properly, which is important for digestion and removing harmful substances from your liver.
How to Use This Medicine
Taking Your Medication Correctly
To ensure you get the most benefit from your medication, follow these steps:
Take your medication exactly as directed by your doctor.
Read all the information provided with your medication and follow the instructions carefully.
Take your medication with food to help your body absorb it properly.
Swallow the capsule whole without chewing or crushing it.
If you have difficulty swallowing the capsule, you can mix the contents with certain foods. For younger children, you can mix the contents with infant formula or breast milk. For older children and adults, you can mix the contents with soft foods like mashed potatoes or apple sauce. To do this:
Open the capsule and mix the contents with the chosen food.
Stir the mixture for 30 seconds.
Swallow the mixture right away.
Do not store the mixture for future use.
Important Interactions with Other Medications
If you take cholestyramine, colesevelam, colestipol, or an antacid that contains aluminum, be sure to take it at the right time. Do not take these medications within 4 to 6 hours before or 1 hour after taking your prescribed medication.
Storing and Disposing of Your Medication
To keep your medication safe and effective:
Store it at room temperature in a dry place.
Do not store it in a bathroom.
Keep all medications out of the reach of children and pets.
Dispose of unused or expired medications properly. Do not flush them down the toilet or pour them down the drain unless instructed to do so.
Check with your pharmacist for guidance on the best way to dispose of your medication. You may also be able to participate in a drug take-back program in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However:
If it is close to the time for your next dose, 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:
Take your medication exactly as directed by your doctor.
Read all the information provided with your medication and follow the instructions carefully.
Take your medication with food to help your body absorb it properly.
Swallow the capsule whole without chewing or crushing it.
If you have difficulty swallowing the capsule, you can mix the contents with certain foods. For younger children, you can mix the contents with infant formula or breast milk. For older children and adults, you can mix the contents with soft foods like mashed potatoes or apple sauce. To do this:
Open the capsule and mix the contents with the chosen food.
Stir the mixture for 30 seconds.
Swallow the mixture right away.
Do not store the mixture for future use.
Important Interactions with Other Medications
If you take cholestyramine, colesevelam, colestipol, or an antacid that contains aluminum, be sure to take it at the right time. Do not take these medications within 4 to 6 hours before or 1 hour after taking your prescribed medication.
Storing and Disposing of Your Medication
To keep your medication safe and effective:
Store it at room temperature in a dry place.
Do not store it in a bathroom.
Keep all medications out of the reach of children and pets.
Dispose of unused or expired medications properly. Do not flush them down the toilet or pour them down the drain unless instructed to do so.
Check with your pharmacist for guidance on the best way to dispose of your medication. You may also be able to participate in a drug take-back program in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However:
If it is close to the time for your next dose, 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 Cholbam exactly as prescribed by your doctor. Do not miss doses.
- Take Cholbam with food to help with absorption and reduce stomach upset.
- If you are taking other medications like bile acid sequestrants (e.g., cholestyramine) or aluminum-based antacids, take Cholbam at least 1 hour before or 4-6 hours after these medications.
- Maintain a healthy diet as recommended by your doctor or dietitian, especially regarding fat intake.
- Regular follow-up appointments and blood tests are crucial to monitor your liver function and the effectiveness of the medication.
Available Forms & Alternatives
Available Strengths:
Dosing & Administration
Adult Dosing
Standard Dose:
10 to 15 mg/kg/day, administered orally once daily or divided twice daily
Dose Range:
10 - 20 mg
Condition-Specific Dosing:
Single enzyme defects in bile acid synthesis:
Initial dose 10 to 15 mg/kg/day, may be increased up to 20 mg/kg/day based on clinical response and serum bile acid levels.
Peroxisomal disorders (e.g., Zellweger spectrum disorders):
Initial dose 10 to 15 mg/kg/day, may be increased up to 20 mg/kg/day based on clinical response and serum bile acid levels.
Pediatric Dosing
Neonatal:
Not established (safety and efficacy not established in neonates <1 month of age)
Infant:
10 to 15 mg/kg/day, administered orally once daily or divided twice daily. May be increased up to 20 mg/kg/day based on clinical response and serum bile acid levels.
Child:
10 to 15 mg/kg/day, administered orally once daily or divided twice daily. May be increased up to 20 mg/kg/day based on clinical response and serum bile acid levels.
Adolescent:
10 to 15 mg/kg/day, administered orally once daily or divided twice daily. May be increased up to 20 mg/kg/day based on clinical response and serum bile acid levels.
Dose Adjustments
Renal Impairment:
Mild:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Moderate:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Severe:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Dialysis:
No specific recommendations; caution and close monitoring advised due to potential for accumulation of bile acids.
Hepatic Impairment:
Mild:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Moderate:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Severe:
No specific dose adjustment recommended, monitor liver function and bile acid levels.
Pharmacology
Mechanism of Action
Cholic acid is a primary bile acid. In patients with single enzyme defects in bile acid synthesis, exogenous cholic acid provides a negative feedback on cholesterol 7-alpha-hydroxylase (CYP7A1), the rate-limiting enzyme in bile acid synthesis, thereby suppressing the production of abnormal, toxic bile acid precursors. It also helps normalize bile flow and fat absorption. In peroxisomal disorders, cholic acid helps replace deficient bile acids and may reduce the accumulation of toxic bile acid intermediates.
Pharmacokinetics
Absorption:
Bioavailability:
Not explicitly quantified for exogenous cholic acid, but well absorbed orally.
Tmax:
Approximately 1-2 hours
FoodEffect:
Food may delay Tmax but does not significantly affect the extent of absorption (AUC).
Distribution:
Vd:
Not available (endogenous substance)
ProteinBinding:
>90%
CnssPenetration:
Limited
Elimination:
HalfLife:
Approximately 2-4 hours (for conjugated bile acids)
Clearance:
Not available (undergoes enterohepatic recirculation)
ExcretionRoute:
Primarily fecal (as conjugated bile acids)
Unchanged:
Negligible
Pharmacodynamics
OnsetOfAction:
Weeks to months (clinical and biochemical response)
PeakEffect:
Not precisely defined, depends on clinical and biochemical normalization
DurationOfAction:
Continuous administration required to maintain 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 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 new or worsening liver problems, including:
+ Dark urine
+ Decreased appetite
+ Upset stomach or stomach pain
+ Light-colored stools
+ Bruising or bleeding
+ Feeling extremely tired
+ Vomiting
+ Yellow skin or eyes
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 or any other unusual symptoms, contact your doctor for advice:
* Diarrhea
Note: This is not an exhaustive list of potential side effects. If you have questions or concerns about side effects, consult 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 new or worsening liver problems, including:
+ Dark urine
+ Decreased appetite
+ Upset stomach or stomach pain
+ Light-colored stools
+ Bruising or bleeding
+ Feeling extremely tired
+ Vomiting
+ Yellow skin or eyes
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 or any other unusual symptoms, contact your doctor for advice:
* Diarrhea
Note: This is not an exhaustive list of potential side effects. If you have questions or concerns about side effects, consult 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:
- Yellowing of your skin or the whites of your eyes (jaundice)
- Dark urine
- Pale or clay-colored stools
- Severe stomach pain
- Nausea or vomiting that doesn't go away
- Unusual tiredness or weakness
- Itching
- Easy bruising or bleeding
Before Using This Medicine
Before taking this medication, it is essential to inform your doctor about the following:
Any allergies you have, including allergies to this drug, any of its components, or other substances, such as foods or medications. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
If you are currently taking cyclosporine, as this may interact with the medication.
All medications you are taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins. This is not an exhaustive list, and it is crucial to disclose all drugs and health problems to your doctor.
Any health problems you have, as these may affect the safety of taking this medication.
It is vital to consult with your doctor and pharmacist to ensure that it is safe to take this medication with all your other drugs and health conditions. Do not initiate, discontinue, or modify the dose of any medication without first consulting your doctor to avoid potential interactions or adverse effects.
Any allergies you have, including allergies to this drug, any of its components, or other substances, such as foods or medications. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
If you are currently taking cyclosporine, as this may interact with the medication.
All medications you are taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins. This is not an exhaustive list, and it is crucial to disclose all drugs and health problems to your doctor.
Any health problems you have, as these may affect the safety of taking this medication.
It is vital to consult with your doctor and pharmacist to ensure that it is safe to take this medication with all your other drugs and health conditions. Do not initiate, discontinue, or modify the dose of any medication without first consulting your doctor to avoid potential interactions or adverse effects.
Precautions & Cautions
Important Information for All Patients Taking This Medication
It is crucial that you inform all of your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
You will need to undergo regular blood tests as directed by your doctor. Be sure to discuss any questions or concerns you have about these tests with your doctor.
If you are pregnant, planning to become pregnant, or are breastfeeding, you must notify your doctor. You and your doctor will need to discuss the potential benefits and risks of this medication to both you and your baby.
Special Considerations for Children
If your child is taking this medication and their weight changes, it is essential to consult with your doctor. The dosage of this medication may need to be adjusted based on your child's weight changes.
It is crucial that you inform all of your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
You will need to undergo regular blood tests as directed by your doctor. Be sure to discuss any questions or concerns you have about these tests with your doctor.
If you are pregnant, planning to become pregnant, or are breastfeeding, you must notify your doctor. You and your doctor will need to discuss the potential benefits and risks of this medication to both you and your baby.
Special Considerations for Children
If your child is taking this medication and their weight changes, it is essential to consult with your doctor. The dosage of this medication may need to be adjusted based on your child's weight changes.
Overdose Information
Overdose Symptoms:
- Increased liver enzyme levels
- Elevated serum bile acid levels
- Diarrhea
- Abdominal discomfort
What to Do:
In case of suspected overdose, contact your doctor or poison control center immediately (1-800-222-1222). Treatment is generally supportive, including monitoring liver function and serum bile acid levels, and managing symptoms.
Drug Interactions
Moderate Interactions
- Bile acid sequestrants (e.g., cholestyramine, colestipol, colesevelam)
- Aluminum-based antacids
Monitoring
Baseline Monitoring
Liver function tests (ALT, AST, GGT, alkaline phosphatase, total and direct bilirubin)
Rationale: To assess baseline liver health and monitor for potential hepatotoxicity or disease progression.
Timing: Prior to initiation of therapy
Serum bile acid levels (total bile acids, specific bile acid precursors)
Rationale: To confirm diagnosis and establish baseline for monitoring therapeutic response.
Timing: Prior to initiation of therapy
Fat-soluble vitamin levels (A, D, E, K)
Rationale: To assess for malabsorption and guide supplementation, as these patients often have deficiencies.
Timing: Prior to initiation of therapy
Prothrombin time/INR
Rationale: To assess Vitamin K status and liver synthetic function.
Timing: Prior to initiation of therapy
Growth parameters (height, weight, head circumference in infants/children)
Rationale: To monitor overall nutritional status and response to therapy in pediatric patients.
Timing: Prior to initiation of therapy
Routine Monitoring
Liver function tests (ALT, AST, GGT, alkaline phosphatase, total and direct bilirubin)
Frequency: Monthly for the first 3 months, then every 3-6 months or as clinically indicated.
Target: Within normal limits or stable baseline.
Action Threshold: Significant elevation (e.g., >2-3x baseline or ULN), especially if accompanied by symptoms; consider dose adjustment or discontinuation.
Serum bile acid levels (total bile acids, specific bile acid precursors)
Frequency: Monthly for the first 3 months, then every 3-6 months or as clinically indicated.
Target: Normalization or significant reduction of toxic bile acid precursors.
Action Threshold: Persistent elevation of toxic bile acids; consider dose adjustment.
Fat-soluble vitamin levels (A, D, E, K)
Frequency: Every 6-12 months or as clinically indicated.
Target: Within normal limits.
Action Threshold: Deficiency; initiate or adjust supplementation.
Prothrombin time/INR
Frequency: Every 6-12 months or as clinically indicated.
Target: Within normal limits.
Action Threshold: Elevation; assess Vitamin K status and liver function.
Growth parameters (height, weight, head circumference in infants/children)
Frequency: Every 3-6 months.
Target: Normal growth velocity for age.
Action Threshold: Growth faltering; assess nutritional intake and disease control.
Symptom Monitoring
- Yellowing of skin or eyes (jaundice)
- Dark urine
- Pale stools
- Severe or persistent abdominal pain
- Unusual tiredness or weakness
- Nausea or vomiting
- Itching
- Easy bruising or bleeding
- Poor weight gain or growth faltering (in children)
Special Patient Groups
Pregnancy
Available data from case reports on cholic acid use in pregnant women are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Use only if the potential benefit justifies the potential risk to the fetus.
Trimester-Specific Risks:
First Trimester:
Insufficient data to assess risk.
Second Trimester:
Insufficient data to assess risk.
Third Trimester:
Insufficient data to assess risk.
Lactation
There are no data on the presence of cholic acid in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Cholbam and any potential adverse effects on the breastfed infant from Cholbam or from the underlying maternal condition. Caution is advised.
Infant Risk:
Unknown; potential for adverse effects on the infant cannot be ruled out.
Pediatric Use
Cholbam is indicated for pediatric patients aged 1 month and older. Dosing is weight-based and individualized. Close monitoring of liver function, bile acid levels, and growth is essential.
Geriatric Use
Clinical studies of Cholbam 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. Monitor liver function closely.
Clinical Information
Clinical Pearls
- Cholbam is an orphan drug used for very rare genetic disorders; diagnosis requires specialized testing.
- Adherence to therapy is critical for preventing progressive liver disease and improving clinical outcomes.
- Dosing is highly individualized and requires careful monitoring of liver function tests and serum bile acid levels.
- Patients often require concomitant fat-soluble vitamin supplementation due to malabsorption.
- The capsules can be opened and the contents mixed with formula, breast milk, or soft food for ease of administration in pediatric patients or those with swallowing difficulties.
Alternative Therapies
- Liver transplantation (for end-stage liver disease not responsive to medical therapy)
- Other bile acids (e.g., ursodiol) are not direct therapeutic alternatives for the specific enzyme deficiencies treated by cholic acid, but are used for other cholestatic liver diseases.
Cost & Coverage
Average Cost:
Extremely high (e.g., $30,000 - $50,000+) per 90 capsules (50mg)
Insurance Coverage:
Specialty Tier (requires prior authorization, often subject to high co-pays or co-insurance)
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's a good idea to check with your pharmacist for more information. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek medical attention. When reporting the incident, be prepared to provide details about the medication taken, the amount, and the time it occurred.