Norursodeoxycholic acid significantly reduced levels of serum alkaline phosphatase with similar rates of adverse events compared to placebo
April 16, 2016, Barcelona, Spain: A new study presented today provides hope for a new treatment in patients with primary sclerosing cholangitis, a rare condition characterised by inflammation and scarring in the bile ducts of the liver and for which there are currently no medicines. The study, presented at The International Liver Congress™ 2016 in Barcelona, Spain, showed that at 12 weeks, Norursodeoxycholic acid significantly reduced levels of serum alkaline phosphatase, an enzyme found in the blood of which levels increase in the presence of liver disease.
In primary sclerosing cholangitis, damage to the bile ducts results in the build-up of bile acids in the liver causing damage to liver tissue.1 Early symptoms include tiredness and itching, however as the disease progresses, it may lead to liver cirrhosis (scarring of the liver), high blood pressure in the vessels between the liver and the gut, liver failure and liver cancer, including bile duct cancer.1 The prevalence of primary sclerosing cholangitis is estimated to be 6.3 cases in 100,000 people in the EU, or approximately 82,000 people, and in advanced cases, patients may require liver transplants.1
“Our study demonstrates that Norursodeoxycholic acid could be a viable treatment option for patients with this chronic and debilitating condition,” said Professor Michael Trauner, Division of Gastroenterology and Hepatology, Medical University Vienna and lead author of the study. “Norursodeoxycholic acid provided a safe and effective option for patients with primary sclerosing cholangitis and the treatment warrants further investigation in a larger-scale Phase 3 trial.”
The multicentre European Phase 2, double-blind randomised trial was conducted in 159 patients. Patients with elevated serum alkaline phosphatase levels were randomised to a 12 week treatment period and a four week follow-up period with any treatment. Patients received either 500mg, 1000mg or 1500mg of Norursodeoxycholic acid daily, or placebo. The primary efficacy endpoint of the study was the mean relative change in serum alkaline phosphatase between the baseline visit and the end of treatment visit in the intention to treat population (ITT).
In the ITT population, 500mg, 1000mg and 1500mg of Norursodeoxycholic acid decreased serum alkaline phosphatase by 12.3% (p=0.0029), 17.3% (p=0.0003), and 26% (p<0.0001) respectively. Adverse events occurred at similar frequencies in all treatment groups and the placebo group. Notably, also severe itching of the skin (pruritus) occurred at low frequencies with only small differences among groups: 7 events in the 500mg/d, 7 events in the 1.000mg/d, 12 events in the 1.500mg/d groups and 10 events in the placebo group.
“This is a rare but life-threatening liver disease affecting mostly young individuals. Norursodeoxycholic acid provides hope for this population, as this could be the first pharmacological treatment option for this disease,” said Professor Frank Tacke, EASL Governing Board member.
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About The International Liver Congress™
This annual congress is the biggest event in the EASL calendar, attracting scientific and medical experts from around the world to learn about the latest in liver research. Attending specialists present, share, debate and conclude on the latest science and research in hepatology, working to enhance the treatment and management of liver disease in clinical practice. This year, the congress is expected to attract approximately 10,000 delegates from all corners of the globe. The International Liver Congress™ takes place from April 13 – 17, 2016, at the Fira Barcelona Gran Via, Barcelona, Spain.
About EASL (www.easl.eu)
Since EASL’s foundation in 1966, this not-for-profit organisation has grown to over 4,000 members from all over the world, including many of the leading hepatologists in Europe and beyond. EASL is the leading liver association in Europe, having evolved into a major European Association with international influence, with an impressive track record in promoting research in liver disease, supporting wider education and promoting changes in European liver policy.
For more information, please contact the ILC Press Office at:
- Email: ILCpressoffice@ruderfinn.co.uk
- Telephone: +44 (0)7841 009 252
Onsite location reference
Late-breaker session, Hall 6.0
Saturday 16 April, 16:00 – 18:00
Presenter: Michael Trauner, Austria
Abstract: LB02, Norursodeoxycholic acid improves cholestasis in primary sclerosing cholangitis: Results of a Phase II dose finding study
Author disclosures of interest
Michael Trauner has received honoraria, research grants and travel support by Falk and is listed as co-inventor on a patent on the medical use of norUDCA
1 Khurana V. Primary Sclerosing Cholangitis. Medscape, 03 January 2016. Available from: http://emedicine.medscape.com/article/187724-overview. Last acceded: March 2016.