In your patients with SLOS, CHOLBAM may help*:
Increase plasma
cholesterol2†
In a 2-month pilot study, 11 of 12 patients had increases in plasma cholesterol ranging from 3.8% to 85.7% (mean 38.7 ± 23.3%).
Lower key liver
enzymes1
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values reduced to less than 50 U/L, or baseline levels reduced by 80%.
Total bilirubin values reduced to less than or equal to 1 mg/dL.
Improve certain growth parameters1-3
In a pivotal study, responders saw a 10% increase in body weight or maintained weight above the 50th percentile.
In a 2-month pilot study, 9 of 12 patients showed an increasing trend in weight gain from baseline, though statistical significance was not reached.
Significant improvements in height have been reported.
Years of Clinical Data and Real-World Use
Backed by >18 years of safety data, CHOLBAM has a well-characterized safety profile. There were 12 adverse reactions reported across 9 patients in the clinical trials.1‡ In clinical trials, the most common adverse reaction (~2%) reported was diarrhea.1
CHOLBAM helps patients absorb what matters.
It has been FDA approved since 2015.1
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
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%)
*No evaluable patients with SLOS were in the pivotal studies.1 Patients had other bile acid synthesis disorders due to single enzyme defects.
†In 11 of 12 patients with SLOS given CHOLBAM 10 mg/kg/day and cholesterol supplementation for 2 months, in an open-label pilot study. Long-term studies are needed to determine durability of effect and possible clinical benefits.2
‡ Clinical safety experience with CHOLBAM consists of1:
- Trial 1: a non-randomized, open-label, single-arm trial of 50 patients with bile acid synthesis disorders due to single enzyme defects (SEDs) and 29 patients with peroxisomal disorders (PDs), including Zellweger spectrum disorders. Safety data are available over the 18 years of the trial
- Trial 2: an extension trial of 12 new patients (10 SED and 2 PD), along with 31 patients (21 SED and 10 PD) who rolled over from Trial 1
Safety data are available for 3 years and 11 months of treatment. Adverse events were not collected systematically in either of these trials. Most patients received an oral dose of 10 to 15 mg/kg/day of CHOLBAM.
§Adverse reactions that occurred in new patients.1
Mirum Access Plus can make starting CHOLBAM easier for you and your patients
CHOLBAM is simple for patients to start
INDICATIONS AND LIMITATIONS OF USE
CHOLBAM® (cholic acid) is a bile acid indicated for
•
Treatment of bile acid synthesis disorders due to single enzyme defects.
•
Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.
LIMITATIONS OF USE
The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment
•
Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment.
•
Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose.
•
Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis.
ADVERSE REACTIONS
•
The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy.
DRUG INTERACTIONS
•
Inhibitors of Bile Acid Transporters: Avoid concomitant use of inhibitors of the bile salt efflux pump (BSEP) such as cyclosporine. Concomitant medications that inhibit canalicular membrane bile acid transporters such as the BSEP may exacerbate accumulation of conjugated bile salts in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitoring of serum transaminases and bilirubin is recommended.
•
Bile Acid Binding Resins: Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the efficacy of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin.
•
Aluminum-based Antacids: Aluminum-based antacids have been shown to adsorb bile acids in vitro and can reduce the bioavailability of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after an aluminum-based antacid.
PREGNANCY
No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM.
LACTATION
Endogenous cholic acid is present in human milk. Clinical lactation studies have not been conducted to assess the presence of CHOLBAM in human milk, the effects of CHOLBAM on the breastfed infant, or the effects of CHOLBAM 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.
GERIATRIC USE
It is not known if elderly patients respond differently from younger patients.
HEPATIC IMPAIRMENT
•
Discontinue treatment with CHOLBAM if liver function does not improve within 3 months of the start of treatment.
•
Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline.
OVERDOSAGE
Concurrent elevations of serum GGT and serum ALT may indicate CHOLBAM overdose. In the event of overdose, the patient should be monitored and treated symptomatically. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline.
Please see full Prescribing Information for additional Important Safety Information.
References: 1. CHOLBAM® (cholic acid) capsules. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Elias ER, Orth LE, Li A, Xu L, Jones SM, Rizzo WB. Cholic acid increases plasma cholesterol in Smith-Lemli-Opitz syndrome: a pilot study. Mol Genet Metab Rep. 2023;38:101030. doi:10.1016/j.ymgmr.2023.101030 3. Ferren EC, Hillman PR, Kritzer A, et al. Use of cholic acid in Smith-Lemli-Opitz Syndrome (SLOS): real-world patient outcomes. Poster presented at: 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting; March 18-22, 2025; Los Angeles, CA.