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For adults with primary biliary cholangitis (PBC)1

For adults with primary biliary cholangitis (PBC)1

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PATIENT CASE STUDIES

Explore the hypothetical patient profiles below to see how OCALIVA can work for a range of patient types.

Kathy, OCALIVA® (obeticholic acid) patient case study

Kathy

Patient With Highly Elevated ALP and Advanced Fibrosis Stage at Baseline

* This is a hypothetical patient profile. Individual patient experiences may vary.

Patient Medical Profile:

  • 52 years of age
  • Diagnosed with PBC 6 months ago
  • Has been on UDCA since diagnosis
  • Complains of bouts of fatigue but states that it is manageable
  • Baseline TE: 10 kPa
  • Her physician is concerned with her elevated ALP levels

VIEW Kathy's PROFILE

At 6 months

Considering that Kathy has advanced fibrosis at baseline (when UDCA is initiated), she returns to her physician's office for a UDCA response assessment at 6 months.

Select Laboratory Values
At UDCA Initiation
(6 months ago)		 Currently
ALP (ULN: 118 U/L) 650 U/L 495 U/L
ALT (ULN: 36 U/L) 71 U/L 60 U/L
AST (ULN: 33 U/L) 60 U/L 50 U/L
Total bilirubin (ULN: 1.1 mg/dL) 1.1 mg/dL 1.0 mg/dL
GGT (ULN: 40 U/L) 310 U/L 225 U/L
Albumin (LLN: 3.5 g/dL) 4.1 g/dL 4.1 g/dL
LDL-C (ULN: 100 mg/dL) 130 mg/dL 132 mg/dL
HDL-C (LLN: 50 mg/dL) 85 mg/dL 79 mg/dL
Total cholesterol (ULN: 200 mg/dL) 240 mg/dL 220 mg/dL
Fibrosis Score
Transient elastography 10 kPa 9 kPa

Upon reviewing the laboratory results, Kathy’s physician is striving for further reduction in her liver biochemistries and decides to start her on OCALIVA 5 mg in addition to the UDCA she has been on for 6 months. To support her during her treatment changes, Kathy’s NP sets up a follow-up phone call 2 weeks after OCALIVA initiation. She reports that treatment is going well, with no new or worsening symptoms or issues.

WHEN WOULD YOU FOLLOW UP WITH KATHY AFTER OCALIVA INITIATION?
WHAT QUESTIONS WOULD YOU EXPECT TO HEAR FROM HER?

At 1-month follow-up

Kathy returns for follow-up 1 month after OCALIVA initiation. Treatment is still going well; no new or worsening symptoms/issues.

Select Laboratory Values
At UDCA Initiation
(7 months ago)		 At OCALIVA Initiation
(1 month ago)		 1 month on OCALIVA
ALP (ULN: 118 U/L) 650 U/L 495 U/L 365 U/L
ALT (ULN: 36 U/L) 71 U/L 60 U/L 38 U/L
AST (ULN: 33 U/L) 60 U/L 50 U/L 32 U/L
Total bilirubin (ULN: 1.1 mg/dL) 1.1 mg/dL 1.0 mg/dL 0.8 mg/dL
GGT (ULN: 40 U/L) 310 U/L 225 U/L 160 U/L
Albumin (LLN: 3.5 g/dL) 4.1 g/dL 4.1 g/dL 4.1 g/dL
LDL-C (ULN: 100 mg/dL) 130 mg/dL 132 mg/dL 135 mg/dL
HDL-C (LLN: 50 mg/dL) 85 mg/dL 79 mg/dL 72 mg/dL
Total cholesterol (ULN: 200 mg/dL) 240 mg/dL 220 mg/dL 210 mg/dL
Fibrosis Score
Transient elastography 10 kPa 9 kPa –

At 3-month follow-up

Kathy returns 2 months later for her next follow-up visit. Treatment is still going well; no new or worsening symptoms/issues.

Select Laboratory Values
At UDCA Initiation
(9 months ago)		 At OCALIVA Initiation
(3 months ago)		 1 month on OCALIVA
5 mg + UDCA		 3 months on OCALIVA
5 mg + UDCA
ALP (ULN: 118 U/L) 650 U/L 495 U/L 365 U/L 315 U/L
ALT (ULN: 36 U/L) 71 U/L 60 U/L 38 U/L 23 U/L
AST (ULN: 33 U/L) 60 U/L 50 U/L 32 U/L 20 U/L
Total bilirubin (ULN: 1.1 mg/dL) 1.1 mg/dL 1.0 mg/dL 0.8 mg/dL 0.8 mg/dL
GGT (ULN: 40 U/L) 310 U/L 225 U/L 160 U/L 130 U/L
Albumin (LLN: 3.5 g/dL) 4.1 g/dL 4.1 g/dL 4.1 g/dL 4.1 g/dL
LDL-C (ULN: 100 mg/dL) 130 mg/dL 132 mg/dL 135 mg/dL 135 mg/dL
HDL-C (LLN: 50 mg/dL) 85 mg/dL 79 mg/dL 72 mg/dL 71 mg/dL
Total cholesterol (ULN: 200 mg/dL) 240 mg/dL 220 mg/dL 210 mg/dL 210 mg/dL
Fibrosis Score
Transient elastography 10 kPa 9 kPa – –

DO YOU FEEL THAT KATHY IS A CANDIDATE FOR UP-TITRATION TO OCALIVA 10 MG?

Lucia, OCALIVA® (obeticholic acid) patient case study

Lucia

Fibrosis Progression and Initiation of Second-Line Therapy

* This is a hypothetical patient profile. Individual patient experiences may vary.

Patient Medical Profile:

  • 43 years of age
  • Diagnosed with PBC 1 year ago
  • Has been on UDCA since diagnosis
  • Social history: Immigrant, non-English speaker
  • ALP elevation
  • Baseline TE: 11 kPa
  • Ultrasound of the spleen: Normal

VIEW Lucia's PROFILE

At 12 months

Select Laboratory Values
At UDCA Initiation
(12 months ago)		 Currently
ALP (ULN: 118 U/L) 342 U/L 197 U/L
ALT (ULN: 36 U/L) 42 U/L 35 U/L
AST (ULN: 33 U/L) 50 U/L 40 U/L
Total bilirubin (ULN: 1.1 mg/dL) 1.0 mg/dL 0.9 mg/dL
GGT (ULN: 40 U/L) 225 U/L 155 U/L
Albumin (LLN: 3.5 g/dL) 4.1 g/dL 4.1 g/dL
Platelets 200 x 109/L 175 x 109/L
LDL-C (ULN: 100 mg/dL) 80 mg/dL 85 mg/dL
HDL-C (LLN: 50 mg/dL) 90 mg/dL 90 mg/dL
Total cholesterol (ULN: 200 mg/dL) 170 mg/dL 172 mg/dL
Fibrosis Score
Transient elastography 11 kPa 12 kPa

At Lucia’s 12-month follow-up after UDCA initiation, her physician is concerned with her fibrosis score. So, they decide to start her on OCALIVA 5 mg in addition to UDCA, and plan to follow up after 3 months.

At 3 months on OCALIVA

Select Laboratory Values
At UDCA Initiation
(18 months ago)		 Follow-Up on UDCA
(6 months ago)		 Currently
(3 months on OCALIVA 5 mg + UDCA)
ALP (ULN: 118 U/L) 342 U/L 197 U/L 190 U/L
ALT (ULN: 36 U/L) 42 U/L 35 U/L 21 U/L
AST (ULN: 33 U/L) 50 U/L 40 U/L 22 U/L
Total bilirubin (ULN: 1.1 mg/dL) 1.0 mg/dL 0.9 mg/dL 0.9 mg/dL
GGT (ULN: 40 U/L) 225 U/L 155 U/L 140 U/L
Albumin (LLN: 3.5 g/dL) 4.1 g/dL 4.1 g/dL 4.1 g/dL
Platelets 200 x 109/L 175 x 109/L 180 x 109/L
LDL-C (ULN: 100 mg/dL) 80 mg/dL 85 mg/dL 84 mg/dL
HDL-C (LLN: 50 mg/dL) 90 mg/dL 90 mg/dL 92 mg/dL
Total cholesterol (ULN: 200 mg/dL) 170 mg/dL 172 mg/dL 175 mg/dL
Fibrosis Score
Transient elastography 11 kPa 12 kPa 12 kPa

While treatment is still going well, with no new or worsening symptoms/issues, Lucia's physician is still concerned about the elevated fibrosis levels and is also striving for further reduction in her liver biochemistries.

Is Lucia a candidate for up-titration to 10 mg? What are safety considerations for the dose increase?

Cindy, OCALIVA® (obeticholic acid) patient case study

Cindy

Pruritus Management Strategies

* This is a hypothetical patient profile. Individual patient experiences may vary.

Patient Medical Profile:

  • 50 years of age
  • Diagnosed with PBC a little over 1 year ago
  • Has been on UDCA since diagnosis
  • Due to persistently elevated liver biochemistries, her physician adds OCALIVA 5 mg to her treatment regimen

VIEW Cindy's PROFILE

Cindy’s physician calls her after 2 weeks of OCALIVA treatment. At that time, Cindy reports more frequent pruritus since OCALIVA initiation. She had bouts of intermittently mild pruritus prior to OCALIVA initiation, but is currently not being medically treated for pruritus.

Her physician prescribes an antihistamine to be taken at bedtime to address the more frequent pruritus.

HOW WOULD YOU ADDRESS CINDY’S CONCERNS?

At 1 month on OCALIVA

Select Laboratory Values
At OCALIVA Initiation
(1 month ago)		 Currently
ALP (ULN: 118 U/L) 282 U/L 245 U/L
ALT (ULN: 36 U/L) 45 U/L 33 U/L
AST (ULN: 33 U/L) 42 U/L 28 U/L
Total bilirubin (ULN: 1.1 mg/dL) 0.5 mg/dL 0.5 mg/dL
GGT (ULN: 40 U/L) 160 U/L 102 U/L
Albumin (LLN: 3.5 g/dL) 4.3 g/dL 4.3 g/dL
LDL-C (ULN: 100 mg/dL) 145 mg/dL 144 mg/dL
HDL-C (LLN: 50 mg/dL) 74 mg/dL 68 mg/dL
Total cholesterol (ULN: 200 mg/dL) 244 mg/dL 237 mg/dL
Transient elastography 8.0 kPa –

At her 1-month follow up, Cindy reports an improvement in her pruritus.

At 3 months

Select Laboratory Values
At OCALIVA Initiation
(3 months ago)		 1 month on OCALIVA
(5 mg + UDCA)		 Currently
ALP (ULN: 118 U/L) 282 U/L 245 U/L 215 U/L
ALT (ULN: 36 U/L) 45 U/L 33 U/L 28 U/L
AST (ULN: 33 U/L) 42 U/L 28 U/L 20 U/L
Total bilirubin (ULN: 1.1 mg/dL) 0.5 mg/dL 0.5 mg/dL 0.5 mg/dL
GGT (ULN: 40 U/L) 160 U/L 102 U/L 87 U/L
Albumin (LLN: 3.5 g/dL) 4.3 g/dL 4.3 g/dL 4.3 g/dL
LDL-C (ULN: 100 mg/dL) 145 mg/dL 144 mg/dL 145 mg/dL
HDL-C (LLN: 50 mg/dL) 74 mg/dL 68 mg/dL 66 mg/dL
Total cholesterol (ULN: 200 mg/dL) 244 mg/dL 237 mg/dL 234 mg/dL
Transient elastography 8.0 kPa – –

And at 3 months, Cindy reported further improvement in her pruritus.

WHAT ADDITIONAL PRURITUS MANAGEMENT TECHNIQUES WOULD YOU CONSIDER FOR CINDY?

HOW COULD OCALIVA CHANGE THE COURSE OF TREATMENT FOR YOUR PATIENTS?

ALP, alkaline phosphatase; ALT, alanine transaminase; AST, aspartate aminotransferase; GGT, gamma-glutamyl transferase; HDL-C, high-density lipoprotein cholesterol; kPa, kilopascal; LDL-C, low-density lipoprotein cholesterol; LLN, lower limit of normal; TE, transient elastography; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.

Reference

  1. OCALIVA full prescribing information. Morristown, NJ: Intercept Pharmaceuticals, Inc; 2022.

INDICATION AND IMPORTANT SAFETY INFORMATION

WARNING: HEPATIC DECOMPENSATION AND FAILURE IN PRIMARY BILIARY CHOLANGITIS PATIENTS WITH CIRRHOSIS

  • Hepatic decompensation and failure, sometimes fatal or resulting in liver transplant, have been reported with OCALIVA treatment in primary biliary cholangitis (PBC) patients with either compensated or decompensated cirrhosis.
  • OCALIVA is contraindicated in PBC patients with decompensated cirrhosis, a prior decompensation event, or with compensated cirrhosis who have evidence of portal hypertension.
  • Permanently discontinue OCALIVA in patients who develop laboratory or clinical evidence of hepatic decompensation; have compensated cirrhosis and develop evidence of portal hypertension; or experience clinically significant hepatic adverse reactions while on treatment.

INDICATION

OCALIVA, a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adult patients with primary biliary cholangitis (PBC)

  • without cirrhosis or
  • with compensated cirrhosis who do not have evidence of portal hypertension,

either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.

This indication is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Contraindications

OCALIVA is contraindicated in patients with:

  • decompensated cirrhosis (e.g., Child-Pugh Class B or C) or a prior decompensation event.
  • compensated cirrhosis who have evidence of portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia).
  • complete biliary obstruction.

Warnings and Precautions

Hepatic Decompensation and Failure in PBC Patients with Cirrhosis

Hepatic decompensation and failure, sometimes fatal or resulting in liver transplant, have been reported with OCALIVA treatment in PBC patients with cirrhosis, either compensated or decompensated. Among postmarketing cases reporting it, median time to hepatic decompensation (e.g., new onset ascites) was 4 months for patients with compensated cirrhosis; median time to a new decompensation event (e.g., hepatic encephalopathy) was 2.5 months for patients with decompensated cirrhosis. Some of these cases occurred in patients with decompensated cirrhosis when they were treated with higher than the recommended dosage for that patient population; however, cases of hepatic decompensation and failure have continued to be reported in patients with decompensated cirrhosis even when they received the recommended dosage.

Hepatotoxicity was observed in the OCALIVA clinical trials. A dose-response relationship was observed for the occurrence of hepatic adverse reactions including jaundice, worsening ascites, and primary biliary cholangitis flare with dosages of OCALIVA of 10 mg once daily to 50 mg once daily (up to 5-times the highest recommended dosage), as early as one month after starting treatment with OCALIVA in two 3-month, placebo-controlled clinical trials in patients with primarily early stage PBC.

Routinely monitor patients for progression of PBC, including hepatic adverse reactions, with laboratory and clinical assessments to determine whether drug discontinuation is needed. Closely monitor patients with compensated cirrhosis, concomitant hepatic disease (e.g., autoimmune hepatitis, alcoholic liver disease), and/or with severe intercurrent illness for new evidence of portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia) or increases above the upper limit of normal in total bilirubin, direct bilirubin, or prothrombin time to determine whether drug discontinuation is needed. Permanently discontinue OCALIVA in patients who develop laboratory or clinical evidence of hepatic decompensation (e.g., ascites, jaundice, variceal bleeding, hepatic encephalopathy), have compensated cirrhosis and develop evidence of portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia), experience clinically significant hepatic adverse reactions, or develop complete biliary obstruction. If severe intercurrent illness occurs, interrupt treatment with OCALIVA and monitor the patient’s liver function. After resolution of the intercurrent illness, consider the potential risks and benefits of restarting OCALIVA treatment.

Severe Pruritus

Severe pruritus was reported in 23% of patients in the OCALIVA 10 mg arm, 19% of patients in the OCALIVA titration arm, and 7% of patients in the placebo arm in a 12-month double-blind randomized controlled clinical trial of 216 patients. Severe pruritus was defined as intense or widespread itching, interfering with activities of daily living, or causing severe sleep disturbance, or intolerable discomfort, and typically requiring medical interventions. Consider clinical evaluation of patients with new onset or worsening severe pruritus. Management strategies include the addition of bile acid binding resins or antihistamines, OCALIVA dosage reduction, and/or temporary interruption of OCALIVA dosing.

Reduction in HDL-C

Patients with PBC generally exhibit hyperlipidemia characterized by a significant elevation in total cholesterol primarily due to increased levels of high-density lipoprotein-cholesterol (HDL-C). Dose-dependent reductions from baseline in mean HDL-C levels were observed at 2 weeks in OCALIVA-treated patients, 20% and 9% in the 10 mg and titration arms, respectively, compared to 2% in the placebo arm. Monitor patients for changes in serum lipid levels during treatment. For patients who do not respond to OCALIVA after 1 year at the highest recommended dosage that can be tolerated (maximum of 10 mg once daily), and who experience a reduction in HDL-C, weigh the potential risks against the benefits of continuing treatment.

Adverse Reactions

The most common adverse reactions (≥5%) are: pruritus, fatigue, abdominal pain and discomfort, rash, oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function abnormality, and eczema.

Drug Interactions

  • Bile Acid Binding Resins
    Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the absorption, systemic exposure, and efficacy of OCALIVA. If taking a bile acid binding resin, take OCALIVA at least 4 hours before or 4 hours after taking the bile acid binding resin, or at as great an interval as possible.
  • Warfarin
    The International Normalized Ratio (INR) decreased following coadministration of warfarin and OCALIVA. Monitor INR and adjust the dose of warfarin, as needed, to maintain the target INR range when co-administering OCALIVA and warfarin.
  • CYP1A2 Substrates with Narrow Therapeutic Index
    Obeticholic acid may increase the exposure to concomitant drugs that are CYP1A2 substrates. Therapeutic monitoring of CYP1A2 substrates with a narrow therapeutic index (e.g., theophylline and tizanidine) is recommended when co-administered with OCALIVA.
  • Inhibitors of Bile Salt Efflux Pump
    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 including taurine conjugate of obeticholic acid in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitor serum transaminases and bilirubin.

Please click here for Full Prescribing Information, including Boxed WARNING.

To report SUSPECTED ADVERSE REACTIONS, contact Intercept Pharmaceuticals, Inc. at 1-844-782-ICPT or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

If you have questions or would like more information about OCALIVA, contact Intercept Medical Information.