Treating peroxisomal biogenesis disorder-Zellweger spectrum disorder (PBD-ZSD)
If your child has been diagnosed with PBD-ZSD, it’s important to understand how bile acid replacement therapy can help improve signs of liver involvement.
CHOLBAM (KOL-BOM) increases cholic acid that is not produced normally in people with PBD-ZSD. This may help to
- Restore the balance of bile acids in the liver and reduce cholestasis.
- Improve bile flow, which helps the liver absorb vitamins and nutrients that the body needs for overall health.
While PBD-ZSD can impact different organs in the body due to defects in bile acid production, CHOLBAM has only been shown to help relieve the signs and symptoms that are related to liver disease.
CHOLBAM is a bile acid replacement medication that is taken by mouth.
CHOLBAM is indicated for:
- Patients with bile acid synthesis disorders due to single enzyme defects.
- Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who show signs and symptoms of liver disease, steatorrhea (fatty stools), or complications from decreased fat-soluble vitamin absorption (A, D, E, K).
The safety and effectiveness of CHOLBAM on symptoms outside of the liver have not been studied.
During treatment
Monitoring liver function
While taking CHOLBAM, periodic lab testing is recommended. Your doctor will monitor liver function every month for the first 3 months, then test less frequently throughout the year. If liver function does not improve within 3 months of starting treatment, or symptoms worsen, your doctor will discontinue CHOLBAM.
How to take CHOLBAM
- CHOLBAM is available in 50 mg and 250 mg capsules.
- The recommended dosage of CHOLBAM is 10 to 15 mg per kg of body weight taken orally once daily, or in 2 divided doses. Do not adjust the dose without first consulting with your child’s or loved one’s doctor.
- The recommended dosage will change as weight changes. Your child’s doctor will adjust as needed, particularly during periods of rapid growth.
- Talk to your child’s doctor about the right dosage for your child and how many capsules should be taken each day.
- Take CHOLBAM with food.
- Take CHOLBAM at least 1 hour before or 4 to 6 hours after taking a bile acid binding resin or an aluminum-based antacid.
- Do not crush or chew the capsules.
For patients who cannot swallow capsules, the capsules can be opened and the contents mixed with either infant formula or expressed breast milk (for younger children), or soft food such as mashed potatoes or apple puree (for older children and adults) in order to mask any unpleasant taste.
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Hold the capsule over the prepared liquid/food, gently twist open, and allow the contents to fall into the liquid/food.
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Mix the entire capsule contents with one or two tablespoons (15 mL to 30 mL) of infant formula, expressed breast milk, or soft food such as mashed potatoes or apple puree.
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Stir for 30 seconds.
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The capsule contents will remain as fine granules in the milk or food and will not dissolve.
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Administer the mixture immediately.
What to expect
When taken regularly, CHOLBAM may help keep the normal balance of bile acids in the liver. That’s why it’s important to take CHOLBAM every day. Avoid missing doses and do not stop therapy without talking with your child’s doctor.
People taking CHOLBAM will need to undergo laboratory testing periodically while on treatment to assess liver function.
Well-Established Safety Profile
CHOLBAM has been FDA approved and available since 2015.
It is used for the treatment of bile acid synthesis disorders (BASDs), which are caused by defects in the enzyme that makes bile acids.
The safety and effectiveness of CHOLBAM on symptoms outside of the liver have not been studied.
As with any medication, there is a possibility of side effects. In clinical studies, the most common side effect in about 2% of patients taking CHOLBAM was diarrhea. All other side effects were less than or equal to 1% of patients.
CHOLBAM patients will need to undergo laboratory testing periodically while on treatment to assess liver function. CHOLBAM may worsen liver function. Call your child’s doctor right away if there is:
- Yellowing of the skin or whites of the eyes.
- Dark or brown (tea-colored) urine.
- Pain on the right side of the stomach.
- Bleeding or bruising that occurs more easily than normal.
- Increased tiredness.
Most Common Adverse Reactions in Trials 1 and 2
Adverse Reactions |
Trial 1 |
Trial 2 |
Overall (%) |
Diarrhea |
1 |
2* |
3 (2%) |
Reflux esophagitis |
1 |
0 |
1 (1%) |
Malaise |
1 |
0 |
1 (1%) |
Jaundice |
1 |
0 |
1 (1%) |
Skin lesion |
1 |
0 |
1 (1%) |
Nausea |
0 |
1* |
1 (1%) |
Abdominal pain |
0 |
1* |
1 (1%) |
Intestinal polyp |
0 |
1* |
1 (1%) |
Urinary tract infection |
0 |
1* |
1 (1%) |
Peripheral neuropathy |
0 |
1 |
1 (1%) |
Discuss with your child’s doctor any side effects that concern you. You can also view the CHOLBAM full Prescribing Information, which is available to healthcare professionals.
How to receive CHOLBAM
Getting started on CHOLBAM is simple through the Mirum Access Plus. Enroll in the support program today by texting ENROLL to 1-833-MAP-4YOU (1-833-627-4968). Or, download and sign the Mirum Access Plus Consent Form to sign up.
WHAT IS CHOLBAM® (cholic acid) capsules?
CHOLBAM is a prescription medicine. It is a bile acid used for
- Treatment of bile acid synthesis disorders (malfunction in the production of bile acid in the liver) due to a lack of a single enzyme responsible for bile acid production.
- Treatment (in addition to other treatments) of peroxisomal disorders (a loss of function in important parts of the cell), including Zellweger spectrum disorders, in patients who show signs or symptoms of liver disease, pale stools due to too much fat, or problems resulting from not absorbing certain vitamins (A, D, E, K).
The safety and effectiveness of CHOLBAM on symptoms outside of the liver have not been studied.
IMPORTANT SAFETY INFORMATION
CHOLBAM may not be right for you. Before taking CHOLBAM, tell your doctor about:
- All health conditions you may have and any medications you may be taking.
- If you are pregnant or plan to become pregnant.
- If you are breastfeeding or plan to breastfeed.
What are the possible side effects of CHOLBAM?
- Serious Side Effects: You will need to undergo laboratory testing periodically while on treatment to assess liver function. CHOLBAM may worsen liver function. Tell your doctor right away if you experience symptoms associated with worsening of liver function (e.g., skin or the whites of eyes turn yellow, urine turns dark or brown [tea colored], pain on the right side of stomach, bleeding or bruising occurs more easily than normal, or increased lethargy). Your doctor will decide if you need to discontinue treatment with CHOLBAM.
- Most Common Side Effects: Diarrhea, acid reflux that damages the lining of the esophagus, discomfort and fatigue, yellowing of skin or eyes, skin lesion, nausea, abdominal pain, formation of growths in the intestinal tract, urinary tract infection, nerve damage causing change in sensation in extremities.
These are not all the possible side effects of CHOLBAM. Tell your doctor if you have any side effect that bothers you or that does not go away.
You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Mirum Pharmaceuticals at 1-855-MRM-4YOU.
Please see accompanying full Prescribing Information for additional Important Safety Information.