CHOLBAM® (cholic acid) capsules may help patients with liver dysfunction due to Zellweger spectrum disorders1
CHOLBAM® is an adjunctive, replacement therapy that prevents the accumulation of the abnormal bile acids that occur in patients who have liver involvement in Zellweger Spectrum Disorders. When there is a more normal balance of bile acids in the liver, patients are able to absorb vitamins from the foods they eat, which may help them gain weight and grow. Bile acid replacement therapy also can help improve liver function.1
Zellweger spectrum disorders may lead to liver dysfunction due to bile acid synthesis defects, and can also lead to disorders in other organs of the body. CHOLBAM® has only been shown to help relieve the signs and symptoms that are related to liver disease.1*
Bile acid replacement therapy also can help improve liver function2
Individual results may vary; not all patients will experience similar levels of response.
*Limitation of Use
The safety and effectiveness of CHOLBAM® on extrahepatic manifestations of peroxisomal disorders including Zellweger spectrum disorders have not been established.1
1. CHOLBAM® (cholic acid) capsules, for oral use [prescribing information]. San Diego, CA: Retrophin, Inc.; March 2015. 2. Heubi JE, Setchell K, Bove KE. Inborn errors of bile acid metabolism. Semin Liver Dis. 2007;27(3):282-294.
Indication and Usage
CHOLBAM® 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 show signs and symptoms of liver disease, steatorrhea (fatty stools), or complications from decreased fat soluble vitamins absorption (A, D, E, K)
Limitation of Use:
The safety and effectiveness of CHOLBAM® on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorder including Zellweger spectrum disorders have not been established.
Important Safety Information
Warnings and Precautions—Exacerbation of Liver Impairment
- Monitor liver function and discontinue CHOLBAM® (cholic acid) in patients who develop worsening of liver function while on treatment.
- Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin, and international normalized ratio (INR) every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the next 3 years and annually thereafter. Administer the lowest dose that effectively maintains liver function.
- Discontinue CHOLBAM® if liver function does not improve within 3 months of starting treatment, if complete biliary obstruction develops, or if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis; continue to monitor liver function and consider restarting at a lower dose when parameters return to baseline.
In the CHOLBAM® clinical trials, diarrhea was the most common adverse reaction in approximately 2% of the patient population. All other adverse reactions are less than or equal to 1% of the patient population.
- Bile Salt Efflux Pump (BSEP) Inhibitors (eg, cyclosporine): Avoid concomitant use; if concomitant use is necessary, monitor serum transaminases and bilirubin.
- Bile Acid Resins and Aluminum-Based Antacids: 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 or aluminum-based antacids.
No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM®. Women who become pregnant during CHOLBAM® treatment are encouraged to call 1-844-202-6262.
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.
There are no animal lactation data and no data from case reports available in the published literature.
In the event of overdose (elevated GGT and ALT), the patient should be monitored and treated symptomatically.
Elevated serum gamma glutamyltransferase (GGT) and serum alanine aminotransferase (ALT) may indicate CHOLBAM® overdose. 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 CHOLBAM® (cholic acid) 50 mg and 250 mg capsules.