Drug updated on 4/26/2024
| Dosage Form | Tablet (oral; 5 mg, 10 mg) |
| Drug Class | Farnesoid X receptor agonists |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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.
Summary
- Obeticholic acid (Ocaliva) is used for treating adult patients with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not exhibit evidence of portal hypertension. It is used either in combination with ursodeoxycholic acid (UDCA) for those with an inadequate response to UDCA, or as a standalone treatment in patients unable to tolerate UDCA.
- Six studies provided a comprehensive analysis of Ocaliva's efficacy and safety profile, focusing on conditions such as PBC and non-alcoholic steatohepatitis (NASH).
- In terms of efficacy, obeticholic acid was effective across multiple noninvasive testing endpoints for fibrosis in NAFLD/NASH. It showed significant histological improvement at both 10mg and 25mg doses but also indicated a dose-dependent increase in adverse events.
- For PBC treatment, obeticholic acid demonstrated superior efficacy over placebo significantly affecting lipid profiles, albeit with an increase in LDL-C levels and a decrease in HDL-C levels. Its beneficial outcomes were notably observed when used in combination with UDCA, especially for individuals showing an inadequate response to UDCA monotherapy.
- Safety concerns include an increased incidence of pruritus among treated individuals, along with significant adverse events at higher doses, such as the 25mg dose used for NASH treatments. Adverse changes in lipid profiles call for careful monitoring.
- Compared to other therapeutic options like semaglutide, firsocostat, and the combination of cilofexor and firsocostat, obeticholic acid consistently showed efficacy but necessitates caution due to potential side effects, including pruritus and detrimental changes in lipid profiles that may require further interventions or therapies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Ocaliva (obeticholic acid) Prescribing Information. | 2022 | Intercept Pharmaceuticals, Inc., New York, NY |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| APASL clinical practice guidance: The diagnosis and management of patients with primary biliary cholangitis. | 2022 | Hepatology International |
| A consensus integrated care pathway for patients with primary biliary cholangitis: A guideline-based approach to clinical care of patients. | 2021 | Expert Review of Gastroenterology & Hepatology |
| Primary biliary cholangitis: 2021 practice guidance update from the American Association for the Study of Liver Diseases. | 2021 | Hepatology |
| Practical strategies for pruritus management in the obeticholic acid-treated patient with PBC: Proceedings from the 2018 expert panel. | 2019 | BMJ Open Gastroenterology |