CHOLBAM may help increase cholesterol levels and improve liver function
Smith-Lemli-Opitz Syndrome (SLOS) may affect the body’s ability to make enough cholesterol. Since some bile acids are made from cholesterol and are needed for absorbing important nutrients from food, people with very low cholesterol may struggle with proper digestion and nutrient absorption.
CHOLBAM may help people*:
Increase plasma cholesterol†
CHOLBAM may help raise the amount of cholesterol in the blood, an essential substance that plays vital roles in the body.
Grow on schedule‡
By improving cholesterol levels, CHOLBAM may help people improve their weight and height. This may help address weight and height delays in people with SLOS.
Lower liver enzymes
CHOLBAM may help lower liver enzymes, such as alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Lowering these enzymes may support better liver function.
*No evaluable patients with SLOS were represented in pivotal clinical trials. Patients had other bile acid synthesis disorders due to single enzyme defects.
†CHOLBAM was studied in a pilot study. Long-term studies are needed to determine durability of effect and possible clinical benefits.
‡In a 2-month pilot study, 9 of 12 patients showed an increasing trend in weight gain from baseline but did not reach statistical significance.
CHOLBAM increases cholic acid, which is one of the primary bile acids that people with SLOS may lack.
CHOLBAM has a well-established safety profile
CHOLBAM helps patients absorb what matters.
It has been FDA approved since 2015.
CHOLBAM is used for the treatment of bile acid synthesis disorders (BASDs), which are caused by defects in an enzyme that makes bile acids. SLOS is a type of BASD.
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.
CHOLBAM is a daily treatment
