A registry for people taking Cholbam® (Cholic Acid)
Retrophin is conducting a registry study to better understand how Cholbam® is used, how well it works, and its safety. Registry studies are carried out to collect information from people who have a particular condition or are taking a certain medicine. This registry is observational, which means that no new treatment or drugs are given to patients as part of the registry. It will not change the way doctors treat your or your child’s condition.
If you sign your child or loved one up for The REPLACE registry:
- Your child’s doctor will ask you questions about your child’s health and use of medicines, including Cholbam®, at each routine visit.
- All information will remain confidential.
- You could make a difference in the lives of people who are prescribed Cholbam® in the future.
If you are considering being a part of The REPLACE registry and would like to speak to a representative for more information, please contact firstname.lastname@example.org .
You can also find more information about this registry at clinicaltrials.gov
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.