Pharmacy Prior Authorization Criteria

Ocaliva (obeticholic acid)

Indications for Prior Authorization

Ocaliva (obeticholic acid)

• For diagnosis of Primary Biliary Cholangitis
  Indicated for the treatment of primary biliary cholangitis (PBC) in adult patients 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.

Criteria

Ocaliva

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s)

• Patient is currently receiving treatment with Ocaliva [E, 6, 7]
AND
• Diagnosis of primary biliary cholangitis (also known as primary biliary cirrhosis)
AND
• One of the following:
• Both of the following:
• Patient has failed to achieve an alkaline phosphatase (ALP) level of less than 1.67 times the upper limit of normal (ULN) after at least 12 consecutive months of treatment with ursodeoxycholic acid (UDCA) (e.g., Urso, Urso Forte, ursodiol) [A,1,4]
• Used in combination with ursodeoxycholic acid (UDCA)
OR
• History of contraindication or intolerance to ursodeoxycholic acid (UDCA) [B,1-3]
AND
• Requested drug will not be used in combination with Iqirvo (elafibranor) or Livdelzi (seladelpar)
AND
• Prescribed by or in consultation with one of the following:
• Hepatologist
• Gastroenterologist
AND
• Patient does not have evidence of advanced cirrhosis (i.e. cirrhosis with current or prior evidence of hepatic decompensation including encephalopathy or coagulopathy) [C,5]
AND
• Patient does not have evidence of portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia) [C,5]

Ocaliva

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Submission of medical records (e.g., laboratory values) documenting a reduction in ALP level from pre-treatment baseline (i.e., prior obeticholic acid therapy) while on therapy
AND
• Requested drug will not be used in combination with Iqirvo (elafibranor) or Livdelzi (seladelpar)
AND
• Patient does not have evidence of advanced cirrhosis (i.e. cirrhosis with current or prior evidence of hepatic decompensation including encephalopathy or coagulopathy) [C,D,1,5]
AND
• Patient does not have evidence of portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia) [C,D,1,5]

P & T Revisions

2024-12-13, 2024-11-13, 2024-11-01, 2024-10-02, 2024-08-16, 2023-10-03, 2022-10-21, 2021-09-09, 2021-07-12, 2020-09-09, 2019-10-02

References

1. Ocaliva Prescribing Information. Intercept Pharmaceuticals, Inc. New York, NY. February 2022.
2. Kowdley KV, Luketic V, Chapman R, et al. A randomized trial of obeticholic acid monotherapy in patients with primary biliary cholangitis. Hepatology. 2018;67(5):1890-902.
3. Nevens F, Andreone P, Mazzella G, et al. A placebo-controlled trial of obeticholic acid in primary biliary cholangitis. The New England Journal of Medicine. 2016;375(7):375:631-43.
4. Lindor K, Bowlus C, Boyer J, Levy C, Mayo M. Primary Biliary Cholangitis: 2018 Practice Guidance from the American Association for the Study of Liver Diseases. Hepatology. 2018.
5. FDA Drug Safety Communication: FDA adds Boxed Warning to restrict use of Ocaliva (obeticholic acid) in primary biliary cholangitis (PBC) patients with advanced cirrhosis. Food and Drug Administration Web site. https://www.fda.gov/drugs/drug-safety-and-availability/due-risk-serious-liver-injury-fda-restricts-use-ocaliva-obeticholic-acid-primary-biliary-cholangitis. Accessed September 2, 2021.
6. Food and Drug Administration. September 13, 2024 Meeting of the Gastrointestinal Drugs Advisory Committee- Minutes. FDA. Published September 13, 2024. Updated October 11, 2024. Accessed November 13, 2024. https://www.fda.gov/media/182630/download
7. Kansteiner F. FDA rejects Intercept's ask for Ocaliva full nod in rare liver disease, but accelerated approval remains. FiercePharma. Published Nov. 12, 2024. Accessed Nov. 13, 2024. https://www.fiercepharma.com/pharma/fda-declines-grant-intercepts-ocaliva-full-approval-rare-liver-disease

---

End Notes

1. The recommended starting dosage of Ocaliva is 5 mg orally once daily for the first 3 months in adult patients without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, who have not achieved an adequate biochemical response to an appropriate dosage of UDCA for at least 1 year (on a stable dosage for at least 3 months) or are intolerant to UDCA. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating Ocaliva, increase to a maximum dosage of 10 mg once daily [1,4].
2. Obeticholic acid (OCA) was also studied as monotherapy in 60 patients with early disease stage PBC, wherein no UDCA use was allowed for at least 3 months before screening in a phase II trial. Additionally, a phase III trial included only 16 (7%) subjects treated with OCA monotherapy. In an analysis of a pooled dataset consisting of Phase 2 and Phase 3 trials, the responder rate for monotherapy at 3 months was 38%, which is similar to the 41% responder rate achieved for the combination therapy (OCA plus UDCA) [1-3].
3. On 5/26/2021, the FDA issued a black box warning restricting the use of Ocaliva in patients having primary biliary cholangitis (PBC) with advanced cirrhosis of the liver because it can cause serious harm. Some PBC patients with cirrhosis who took Ocaliva, especially those with evidence of advanced cirrhosis, developed liver failure, sometimes requiring liver transplant. In the five years since Ocaliva’s accelerated approval, FDA identified 25 cases of serious liver injury leading to liver decompensation or liver failure associated with Ocaliva in PBC patients with cirrhosis, both in those without clinical signs of cirrhosis (compensated) or in those with clinical signs of cirrhosis (decompensated). Many of these PBC patients had advanced cirrhosis before starting Ocaliva [5].
4. Patients should permanently discontinue Ocaliva if they 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 [1].
5. The Gastrointestinal Drugs Advisory Committee met on September 13, 2024, to discuss whether the supplemental new drug application submitted by Intercept Pharmaceuticals was sufficient to fulfill the accelerated approval postmarketing requirements. The review was extended beyond the initial PDUFA date of Oct. 15, 2024, due to concerns of the reliability of the data from the post-marketing study and observational study, which did not demonstrate clinical benefit, as well as inadequate data to demonstrate the safety of Ocaliva [6]. On Nov. 13, 2024, the FDA issued a complete response letter to Intercept denying full approval of Ocaliva for PBC [7].

---

Revision History

• 2024-12-13: Addition of criteria to reflect FDA's recent denial of full approval based on insufficient data. Currently still available through accelerated approval.
• 2024-11-13: Addition of criteria to reflect FDA's recent denial of full approval based on insufficient data. Currently still available through accelerated approval.
• 2024-11-01: 2024 Annual review - no criteria changes.
• 2024-10-02: Update to initial and reauth criteria to require that medication will not be used in combination with Livdelzi.
• 2024-08-16: Update to initial and reauth criteria to require that medication will not be used in combination with Iqirvo
• 2023-10-03: Annual review - no criteria changes
• 2022-10-21: Annual Review - no criteria updates
• 2021-09-09: 2021 UM Annual Review
• 2021-07-12: Additional criteria to address Black Box Warning
• 2020-09-09: 2020 UM Annual Review.
• 2019-10-02: 2019 Annual Review. SL 10.02.19

---