When it comes to fat-soluble vitamins, nutrients, weight, and growth…
Not an actual patient.
CHOLBAM® (cholic acid) is a bile acid indicated for the treatment of bile acid synthesis disorders (BASDs) due to single enzyme defects and 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.1
CHOLBAM may help patients with BASD due to single enzyme defects.1* Cholic acid may help restore normal liver function by:
Acting through the farnesoid X receptor (FXR) to maintain bile acid homeostasis under normal physiologic conditions1†
Preventing accumulation of hepatotoxic atypical bile acids that may lead to liver damage2-4‡
*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.
† The mechanism of action of cholic acid has not been fully established; however, it is known that cholic acid and its conjugates are endogenous ligands of the nuclear receptor, FXR. FXR regulates enzymes and transporters that are involved in bile acid synthesis and in the enterohepatic circulation to maintain bile acid homeostasis under normal physiologic conditions.1
‡ Response to CHOLBAM treatment was assessed using the following laboratory criteria: (1) alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values reduced to less than 50 U/L, or baseline levels reduced by 80%; (2) total bilirubin values reduced to less than or equal to 1 mg/dL; and (3) no evidence of cholestasis on liver biopsy.1
Stay Up to Date
To receive more information about CHOLBAM as it becomes available, please fill out this form.
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. Gonzales E, Gerhardt MF, Fabre M, et al. Oral cholic acid for hereditary defects of primary bile acid synthesis: a safe and effective long-term therapy. Gastroenterology. 2009;137(4):1310-1320. doi: 10.1053/j.gastro.2009.07.043 3. Berendse K, Klouwer FCC, Koot BGP, et al. Cholic acid therapy in Zellweger spectrum disorders. J Inherit Metab Dis. 2016;39(6):859-868. doi: 10.1007/s10545-016-9962-9 4. Heubi JE, Bove KE, Setchell KDR. Oral cholic acid is efficacious and well tolerated in patients with bile acid synthesis and Zellweger spectrum disorders. J Pediatr Gastroenterol Nutr. 2017;65(3):321-326. doi: 10.1097/MPG.0000000000001657