Odevixibat
Treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged six months and older.
Decisions on record
- Meeting Mar 2026 Not recommended Alagille syndrome (ALGS)
- Meeting Jul 2025 Recommended Progressive familial intrahepatic cholestasis no PSD
- Meeting Mar 2025 Deferred Progressive familial intrahepatic cholestasis (PFIC)
- Meeting Jul 2024 Not recommended Progressive familial intrahepatic cholestasis
Access path
- Jul 2024Not recommended
vs standard of care including partial external biliary…
- ↻ resubmittedMar 2025Recommended · restricted
Comparator changed: standard of care including partial external biliary diversion and…
- Mar 2026Not recommended
Comparator changed: standard of care (SoC) including partial external biliary diversion…
From the public summary
7.1 The PBAC deferred making a recommendation for odevixibat for the treatment of progressive familial intrahepatic cholestasis (PFIC). The PBAC requested draft restrictions which considered revisions to the following aspects: prescriber type for adults, instrument used for assessing pruritus, and criteria for ceasing treatment if no response, for retreatment and for dose escalation.PSD · Mar 2025
7.2 The PBAC recognised that there is a high clinical need for treatment of this ultra rare condition. The PBAC noted the outcomes from the Facilitated Workshop which was held in September 2024. The PBAC noted that there was significant patient and clinician input at the Workshop relating to the symptoms of disease and the proposed patient population and PBS restriction criteria.PSD · Mar 2025
6.33 The resubmission presented a cost-utility analysis (CUA) of odevixibat compared to SoC. The overall structure of the CUA model considered by the PBAC at its July 2024 meeting was unchanged, though the resubmission used different inputs and assumptions to address several issues raised by the PBAC.PSD · Mar 2025
6.34 The key components of the economic evaluation are presented in Table 12.PSD · Mar 2025
Non-Inferiority in terms of safety Source: Table 1-2, p11 of the resubmission a Defined as an average ≥1 grade improvement in pruritus score on the Prucision™ ObsRO Pruritus Scale, assessed over a one-week period prior to dose modification.PSD · Mar 2025
1.3 The timing for dose escalation was reduced from three months without adequate clinical response in the July 2024 submission, to after one month in the resubmission, which was consistent with the Facilitated Resolution Pathway Workshop discussion.PSD · Mar 2025
6.2 The PBAC noted and welcomed the input from individuals (1), health care professionals (4) and organisations (2) via the Consumer Comments facility on the PBS website associated with this resubmission. The PBAC also recalled that the July 2024 submission received input from individuals (35), health care professionals (4) and organisations (3). The comments described a range of benefits of treatment with odevixibat.PSD · Mar 2025
6.3 The PBAC noted the advice received from PFIC Network, PFIC and Related Disorders Australia, and Liver Foundation stating that PFIC often results in a “horrendous itch” that, in some cases may be incessant and lead to continuous scratching and bleeding, lack of sleep, anxiety, depression, post-traumatic stress disorder, and ultimately poor quality of life for patients, as well as their families.PSD · Mar 2025
Cost-effectiveness
ICER value redacted/commercially sensitive; not stated in public summary document
Decision context
PopulationPatients with cholestatic pruritus in Alagille syndrome (ALGS) aged six months or older, with elevated serum bile acids, experiencing itch with average pruritus score ≥2 on Prucision ObsRO Pruritus Scale.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Not recommended | standard of care (SoC) including biliary diversion surgery (SBD) and liver transplant (LT) | — | RCT |
| Mar 2025 | Recommended · restricted | standard of care (SoC) including partial external biliary diversion (PEBD) and liver transplantation (LT) | — | RCT · Pruritus response and serum bile acid response |
| Jul 2024 | Not recommended | standard of care including partial external biliary diversion and liver transplantation | — | RCT · Surrogate |
Clinical evidence
Consumer voice
Consumer input from individuals, healthcare professionals, and patient organisations described significant benefits of odevixibat treatment including improved quality of life with reduced itch, improved sleep, reduced anxiety, and increased ability to attend school and work. Patient organisations emphasised that PFIC causes severe itch leading to poor quality of life, job losses, and inability to
2 months after the first dose, we almost forgot about the itching Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with elevated serum bile acids AND pruritus score ≥2; TGA label has no such biomarker/symptom requirements.