ElafibranorIqirvo
Primary biliary cholangitis (PBC) in patients with inadequate response to ursodeoxycholic acid (UDCA) or intolerance to UDCA, in combination with UDCA or as monotherapy.
Decision on record
- Meeting Mar 2025 Recommended Primary biliary cholangitis (PBC)
Access path
- TGA registered · Iqirvo
TGA label narrower than the PBS population
- Mar 2025Recommended
vs obeticholic acid (OCA)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended elafibranor for the treatment of primary biliary cholangitis (PBC). The PBAC considered that elafibranor was non-inferior in terms of effectiveness and safety compared to obeticholic acid (OCA) and therefore considered that a cost- minimisation approach versus OCA to be appropriate. The PBAC considered that a price premium for elafibranor would be reasonable given the potential reduction in PBC-related pruritus compared to OCA.PSD · Mar 2025
7.2 The PBAC acknowledged the consumer input supporting the submission. The PBAC also noted the input provided during the Sponsor hearing, which highlighted the need for alternative treatments for patients who do not respond to first-line treatment with ursodeoxycholic acid (UCDA). The clinician noted that uptake of OCA in these patients has been lower than expected as it is associated with increased pruritus and other adverse events.PSD · Mar 2025
6.55 The submission presented a modelled economic evaluation based on an indirect comparison of RCTs.PSD · Mar 2025
6.56 The submission did not present economic evidence for elafibranor as a monotherapy for UDCA intolerant patients or as third-line treatment in OCA non-responders separately. The PSCR provided a revised economic model with placebo +/- UDCA as an additional comparator. This was not considered as, being provided with the PSCR, it was not evaluated.PSD · Mar 2025
Elafibranor is superior in terms of safety compared with OCA. Source: Table 1-1, p13 of the submission.PSD · Mar 2025
ALP= alkaline phosphatase; OCA= obeticholic acid; PBC= primary biliary cholangitis; UDCA= ursodeoxycholic acid; WI-NRS= Worst Itch Numeric Rating Scale.PSD · Mar 2025
6.2 The PBAC noted and welcomed the input from an individual (1) and an organisation (1) via the Consumer Comments facility on the PBS website. The comment supported the listing of elafibranor on the PBS.PSD · Mar 2025
6.3 The PBAC noted the advice received from the Liver Foundation which described PBC and its symptoms. The Liver Foundation noted the impact of PBC on quality of life and highlighted the impact of pruritis and fatigue on patients. The Liver Foundation supported the listing of elafibranor on the PBS.PSD · Mar 2025
Cost-effectiveness
ICER value not stated in the public summary document.
Decision context
PopulationAdults aged 18 years or over with primary biliary cholangitis who have experienced inadequate response to UDCA (despite treatment for at least 52 weeks at therapeutic dose) or intolerance to UDCA, with elevated alkaline phosphatase (≥1.67 times ULN with ≥15% reduction from baseline) and bilirubin levels ≤2 times ULN.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended | obeticholic acid (OCA) | — | RCT · Surrogate |
Clinical evidence
Consumer voice
An individual and an organisation (Liver Foundation) provided consumer input supporting the listing of elafibranor on the PBS. The Liver Foundation highlighted the significant impact of primary biliary cholangitis on quality of life, particularly regarding pruritus and fatigue symptoms.
The Liver Foundation noted the impact of PBC on quality of life and highlighted the impact of pruritis and fatigue on patients. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to second-line use with specific biochemical criteria (ALP ≥1.67× ULN, bilirubin ≤2× ULN, 52-week prior UDCA trial) absent from TGA label.