Last reviewed · How we verify
Tauroursodeoxycholic Acid
Tauroursodeoxycholic acid (TUDCA) is a bile acid that reduces endoplasmic reticulum stress and promotes cell survival by stabilizing mitochondrial function and inhibiting apoptosis pathways.
Tauroursodeoxycholic acid (TUDCA) is a bile acid that reduces endoplasmic reticulum stress and promotes cell survival by stabilizing mitochondrial function and inhibiting apoptosis pathways. Used for Progressive familial intrahepatic cholestasis (PFIC), Cerebrotendinous xanthomatosis (CTX), Neurodegenerative diseases (investigational).
At a glance
| Generic name | Tauroursodeoxycholic Acid |
|---|---|
| Also known as | Taurolite, TUDCA, Tudcabil, TUDCA,Tudcabil, Taurolite, taurolite |
| Sponsor | Johns Hopkins University |
| Drug class | Bile acid derivative / Chemical chaperone |
| Target | Endoplasmic reticulum stress pathways; TGR5 receptor (partial) |
| Modality | Small molecule |
| Therapeutic area | Neurology / Rare genetic disorders |
| Phase | FDA-approved |
Mechanism of action
TUDCA is a conjugated bile acid that acts as a chemical chaperone, helping to restore proper protein folding in the endoplasmic reticulum and reducing ER stress-induced cell death. It also has anti-inflammatory and antioxidant properties, protecting cells from mitochondrial dysfunction and apoptosis. These mechanisms make it potentially beneficial in neurodegenerative diseases and other conditions characterized by cellular stress and protein misfolding.
Approved indications
- Progressive familial intrahepatic cholestasis (PFIC)
- Cerebrotendinous xanthomatosis (CTX)
- Neurodegenerative diseases (investigational)
Common side effects
- Diarrhea
- Abdominal discomfort
- Nausea
Key clinical trials
- Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
- TUDCA as a Therapy for Ulcerative Colitis (UC) (PHASE1)
- Integrative Liver-Targeted Therapy for Diabetic Macular Edema: Combining Tauroursodeoxycholate and Traditional Chinese Medicine. (PHASE2)
- Effect of Oral Supplement on Influenza Vaccine Long-term Response
- A Platform Trial for Personalized and Adaptive Therapies in Hepatocellular Carcinoma (PHASE2)
- Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial (PHASE2)
- Tauroursodeoxycholic Acid (TUDCA) Plus Camrelizumab and Regorafenib in Hepatocellular Carcinoma Previously Treated With Anti-PD1/PD-L1 and Bevacizumab (PHASE2)
- Open Label Extension Study of AMX0035 in Patients With ALS (PHASE2)
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:
- Tauroursodeoxycholic Acid CI brief — competitive landscape report
- Tauroursodeoxycholic Acid updates RSS · CI watch RSS
- Johns Hopkins University portfolio CI