Starting Treatment

What are CHOLBAM® (cholic acid) capsules?

CHOLBAM® (KOL-BOM) is a bile acid replacement medication that is taken by mouth.

CHOLBAM® is indicated for

  • Patients with bile acid synthesis disorders due to single enzyme defects (Learn how CHOLBAM® helps)
  • 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 vitamin absorption (A, D, E, K) (Learn how CHOLBAM® helps)

CHOLBAM® is available in 50 mg and 250 mg capsules.

FDA Approved CHOLBAM® FDA Approved CHOLBAM®

Number of capsules needed each day

  • The recommended dosage of CHOLBAM® is 10 to 15 mg/kg administered orally once daily, or in two divided doses, in pediatric and adult patients.
  • The recommended dosage of CHOLBAM® in patients with concomitant familial hypertriglyceridemia is 11 to 17 mg/kg orally once daily, or in two divided doses.

Instructions

  • Take CHOLBAM® with food
  • Take CHOLBAM® at least 1 hour before or 4 to 6 hours after taking a bile acid binding resin or an aluminum-based antacid
  • Do not crush or chew the capsules
  • For patients who cannot swallow capsules, the capsules can be opened and the contents mixed with either infant formula or expressed breast milk (for younger children), or soft food such as mashed potatoes or apple puree (for older children and adults) in order to mask any unpleasant taste:
    1. Hold the capsule over the prepared liquid/food, gently twist open, and allow the contents to fall into the liquid/food.
    2. Mix the entire capsule contents with one or two tablespoons (15 mL to 30 mL) of infant formula, expressed breast milk, or soft food such as mashed potatoes or apple puree.
    3. Stir for 30 seconds.
    4. The capsule contents will remain as fine granules in the milk or food, and will not dissolve.
    5. Administer the mixture immediately.
Mixing contents of capsule into food

It is important to take CHOLBAM® every day
When taken regularly, CHOLBAM® can help keep the normal balance of bile acids in the liver. Avoid missing doses and do not stop therapy without talking with your doctor.


Remember, CHOLBAM® needs to be taken every day

Number of CHOLBAM® Capsules Needed
to Achieve a Recommended Dosage of 10 mg/kg/day

Kg to lbs metric conversion 1kg = 2.2 lbs. Do not adjust the dose without first consulting your physician.

  10 mg/kg/day Dosage
Body Weight (kg) Number of 50 mg capsules Number of 250 mg capsules
4 to 6 1 0
7 to 10 2 0
11 to 15 3 0
16 to 20 4 0
21 to 25 0 1
26 to 30 1 1
31 to 35 2 1
36 to 40* 3 1

Number of CHOLBAM® Capsules Needed
to Achieve a Recommended Dosage of 15 mg/kg/day

Kg to lbs metric conversion 1kg = 2.2 lbs. Do not adjust the dose without first consulting your physician.

  15 mg/kg/day Dosage
Body Weight (kg) Number of 50 mg capsules Number of 250 mg capsules
4 to 5 1 0
6 to 9 2 0
10 to 13 3 0
14 to 16 4 0
17 to 19 0 1
20 to 23 1 1
24 to 26 2 1
27 to 29 3 1
30 to 33 4 1
34 to 36 0 2
37 to 39 1 2
40 to 43* 2 2

*For additional weights, please see dosing charts in accompanying Prescribing Information.

CHOLBAM® (cholic acid) capsules, for oral use [prescribing information]. San Diego, CA: Retrophin, Inc.; March 2015.

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.

Adverse Reactions
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.

Drug Interaction

  • 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.

Pregnancy
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.

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.

There are no animal lactation data and no data from case reports available in the published literature.

Overdosage
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.

To report SUSPECTED ADVERSE REACTIONS, contact Retrophin, Inc. at 1-877-659-5518 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.