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September 09, 2023
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‘Ask me about PBC’ campaign calls on patients to speak up for rare liver disease

Fact checked byRobert Stott
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International Primary Biliary Cholangitis Day, celebrated on September 10, aspires to raise awareness of the challenges that patients with this rare liver disease face and how their stories can help advance diagnosis, treatment and care.

A relatively rare disease, both in the United States and worldwide, PBC affects an estimated 65 out of the 100,000 women in the U.S., according to the American Liver Foundation, with symptoms such as pruritis, fatigue and jaundice presenting later in life, most often in patients aged 45 to 65 years. Although there is currently no cure for PBC, medications are available for disease management and to curb progression of liver damage. The PBC Foundation, through a conference each year, promotes International PBC Day with a different campaign. This year’s campaign — “Ask me about PBC” — is a call for individuals living with PBC to educate others about the rare autoimmune liver condition and, in turn, feel empowered by doing so.

international pbc day

In honor of International PBC Day, Healio has gathered a list of the latest research on treatments for PBC including use of seladelpar, elafibranor, linerixibat and obeticholic acid in combination with bezafibrate.

Q&A: ‘Normalization’ of biochemical markers in PBC may be possible with seladelpar

After 3 months of treatment, seladelpar significantly improved pruritus and markers of liver injury compared with placebo in patients with primary biliary cholangitis, CymaBay Therapeutics recently announced.

According to a company press release, the phase 3 ENHANCE trial assessed the efficacy and safety of seladelpar (CymaBay Therapeutics), an orally active peroxisome proliferator-activated receptor delta agonist, or delpar, in patients with primary biliary cholangitis. The study enrolled patients who were inadequate responders or intolerant to first-line treatment and randomly assigned them to oral daily seladelpar 5 mg (n = 56) or 10 mg (n = 55) or placebo (n = 56). Read more.

VIDEO: ‘Clear correlation’ between IL-31, pruritus in primary biliary cholangitis

In a Healio video exclusive, Andreas Kremer, MD, PhD, MHBA, highlights recent data on primary biliary cholangitis presented at EASL Congress, including risk factors for disease progression and seladelpar for treatment of pruritus.

In a retrospective analysis, Kremer and colleagues examined 1,111 patients with primary biliary cholangitis (PBC) who received ursodeoxycholic acid as first-line treatment. Overall, 26% of patients screen-failed due to elevated alkaline phosphatase levels. Watch here.

‘Encouraging’ ELATIVE data for elafibranor may spark race for primary biliary cholangitis

Topline findings from the phase 3 ELATIVE trial showed that elafibranor improved cholestasis response in patients with primary biliary cholangitis, Ipsen and Genfit announced.

Elafibranor, an investigational dual peroxisome activated receptor agonist, was granted breakthrough therapy designation by the FDA in 2019 for adults with primary biliary cholangitis disease who are intolerant, or have had an inadequate response, to ursodeoxycholic acid (UDCA). Read more.

Ocaliva plus bezafibrate clears FDA orphan drug hurdle for primary biliary cholangitis

The FDA granted Intercept Pharmaceuticals orphan drug designation for the fixed-dose combination of obeticholic acid and bezafibrate intended for treatment of patients with Obeticholic acid (Ocaliva, Intercept Pharmaceuticals) — a farnesoid X receptor agonist — was originally FDA approved for primary biliary cholangitis (PBC) in 2016 in combination with ursodeoxycholic acid. The new regimen pairs obeticholic acid with bezafibrate, a peroxisome proliferator-activated receptor agonist. Read more.

Linerixibat may relieve itch in primary biliary cholangitis-associated pruritus

Linerixibat induced a dose-dependent reduction in itch among patients with primary biliary cholangitis-associated pruritus, according to results published in Clinical Gastroenterology and Hepatology.

“Pruritus is commonly associated with primary biliary cholangitis, present in nearly 75% of patients over the disease course,” Cynthia Levy, MD,FAASLD, AGAF, of the University of Miami Miller School of Medicine, and colleagues wrote. “Pruritus substantially impairs health-related quality of life, affecting sleep and contributing to fatigue.” Read more.

Obeticholic acid yields fewer deaths, liver transplants in primary biliary cholangitis

Patients who received obeticholic acid for primary biliary cholangitis experienced greater transplant-free survival compared with external control patients, according to research published in Gastroenterology.

“This important study is the first to demonstrate that initiating treatment with obeticholic acid (OCA) in appropriate patients with primary biliary cholangitis (PBC) appears to have a meaningful impact on clinical outcomes,” Gideon Hirschfield, FRCP, PhD, Lily and Terry Horner Chair in autoimmune liver disease at the University of Toronto, said in a press release. Read more..

VIDEO: Seladelpar may improve transplant-free survival in primary biliary cholangitis

In a Healio video exclusive, Bettina E. Hansen, MSc, PhD, discusses improvement in transplant-free survival in patients treated with seladelpar for primary biliary cholangitis.

Hansen and colleagues conducted a long-term, open-label study and used the GLOBE score, a risk assessment tool, to determine the effect of oral seladelpar on transplant-free survival in patients with PBC.

Watch here.

FDA grants fast track designation to CNP-104 for treatment of primary biliary cholangitis

Biotechnology company COUR Pharmaceuticals announced that the FDA granted fast track designation to CNP-104, an investigational therapy for the treatment of primary biliary cholangitis, according to a company press release.

“The fast track process is designed to facilitate development for patients with current limited options,” John Puisis, COUR CEO, told Healio. “This designation facilitates early and frequent communication between the FDA and COUR to quickly resolve any questions or issues and can lead to earlier drug approvals and faster access for patients. Read more.

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