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A4250 (odevixibat)
A4250 (odevixibat) is a Bile acid transporter inhibitor Small molecule drug developed by Albireo. It is currently in Phase 3 development for Progressive familial intrahepatic cholestasis type 1 (PFIC1), Bile acid diarrhea.
Odevixibat inhibits the ileal bile acid transporter (IBAT) to reduce bile acid reabsorption and increase fecal excretion, thereby lowering serum bile acids.
Odevixibat inhibits the ileal bile acid transporter (IBAT) to reduce bile acid reabsorption and increase fecal excretion, thereby lowering serum bile acids. Used for Progressive familial intrahepatic cholestasis type 1 (PFIC1), Bile acid diarrhea.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | A4250 (odevixibat) |
|---|---|
| Sponsor | Albireo |
| Drug class | Bile acid transporter inhibitor |
| Target | IBAT (ileal bile acid transporter; SLC10A2) |
| Modality | Small molecule |
| Therapeutic area | Hepatology / Gastroenterology |
| Phase | Phase 3 |
Mechanism of action
By blocking IBAT (also known as SLC10A2), odevixibat prevents the reabsorption of bile acids in the terminal ileum, forcing them to be excreted in feces. This reduces the enterohepatic circulation of bile acids, leading to decreased serum bile acid levels. The mechanism is designed to alleviate pruritus and other symptoms associated with cholestatic liver diseases where bile acid accumulation causes tissue damage and itching.
Approved indications
- Progressive familial intrahepatic cholestasis type 1 (PFIC1)
- Bile acid diarrhea
Common side effects
- Pruritus
- Diarrhea
- Abdominal pain
- Fatigue
Key clinical trials
- Retrospective Observational Study of Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
- Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) (PHASE3)
- Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (PHASE3)
- Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (PHASE3)
- An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (PHASE3)
- This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2 (PHASE3)
- Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
- Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- A4250 (odevixibat) CI brief — competitive landscape report
- A4250 (odevixibat) updates RSS · CI watch RSS
- Albireo portfolio CI
Frequently asked questions about A4250 (odevixibat)
What is A4250 (odevixibat)?
How does A4250 (odevixibat) work?
What is A4250 (odevixibat) used for?
Who makes A4250 (odevixibat)?
What drug class is A4250 (odevixibat) in?
What development phase is A4250 (odevixibat) in?
What are the side effects of A4250 (odevixibat)?
What does A4250 (odevixibat) target?
Related
- Drug class: All Bile acid transporter inhibitor drugs
- Target: All drugs targeting IBAT (ileal bile acid transporter; SLC10A2)
- Manufacturer: Albireo — full pipeline
- Therapeutic area: All drugs in Hepatology / Gastroenterology
- Indication: Drugs for Progressive familial intrahepatic cholestasis type 1 (PFIC1)
- Indication: Drugs for Bile acid diarrhea
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing