CHOLBAM may help support liver function in people with peroxisomal biogenesis disorder-Zellweger spectrum disorder (PBD-ZSD)
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.
Treating PBD-ZSD with CHOLBAM
CHOLBAM increases cholic acid levels, which are often low in people with PBD-ZSD. This may help:
Absorb more fats and fat-soluble vitamins, which may increase certain nutrients.
Restore bile acid balance in the liver, reducing liver blockage (cholestasis)
Improve bile flow, allowing the liver to absorb more important vitamins and nutrients for better 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 lessen the signs and symptoms that are related to liver disease.
CHOLBAM has a well-established safety profile
CHOLBAM helps patients absorb what matters.
It has been FDA approved since 2015.
CHOLBAM is used along with other medications for the treatment of bile acid synthesis disorders (BASDs), which are caused by defects in an 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 1% of patients.
Patients taking CHOLBAM will need to undergo laboratory testing periodically while on treatment to assess liver function. CHOLBAM may worsen liver function.
Call your or your loved one's doctor right away if any of the following occur, as these may be signs of worsening liver function:
- 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%)
*Adverse reactions that occurred in new patients.
Discuss with your or your loved one's doctor any side effects that concern you. You can also view the CHOLBAM full Prescribing Information for additional details.
Talk to a health care provider to learn more about CHOLBAM.
