Hepatology Month in Review: October 2024

As 2024 begins to draw to a close, October was a fitting continuation to what has been a busy year in hepatology. This month, even in the absence of US Food and Drug Administration (FDA) approvals, the hepatic pipeline was as active as ever with a pair of Breakthrough

Therapy Designations for volixibat and survodutide and an extended review of a supplemental New Drug Application for obeticholic acid (Ocaliva).

Additionally, with 2030 looming around the corner, a growing body of research is exploring potential strategies for viral hepatitis treatment and prevention in order to meet the World Health Organization’s goal for elimination. Hepatitis was one of many hepatic topics at the forefront of several posters, abstracts, and sessions at the American College of Gastroenterology (ACG) 2024 Annual Scientific Meeting in Philadelphia, Pennsylvania, this month.

Check out this HCPLive October 2024 hepatology month in review for highlights of our coverage of the biggest hepatic headlines from the past few weeks!

Hepatic Pipeline Updates

FDA Grants Breakthrough Therapy Designation to Survodutide for Noncirrhotic MASH

Early in the month, Boehringer Ingelheim announced the FDA granted Breakthrough Therapy Designation to survodutide (BI 456906), a dual glucagon/glucagon-like peptide-1 (GLP-1) receptor agonist, for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and moderate or advanced fibrosis.

In addition to the Breakthrough Therapy Designation, which was based on phase 2 data published earlier this year, Boehringer also announced the initiation of a pair of phase 3 clinical trials of survodutide for the treatment of adults living with MASH and fibrosis as well as those living with compensated MASH cirrhosis, LIVERAGE and LIVERAGE-Cirrhosis.

FDA Grants Breakthrough Therapy Designation to Volixibat for Cholestatic Pruritus in PBC

Just 2 days later, the FDA granted Breakthrough Therapy Designation to Mirum Pharmaceuticals’ volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC) based on a positive interim analysis of the phase 2b VANTAGE study demonstrating statistically significant improvement versus placebo in pruritus for patients treated with volixibat.

“Breakthrough Therapy Designation for volixibat in PBC underscores the importance and urgency for a treatment to address one of the most burdensome impacts of this rare liver disease,” Joanne Quan, MD, chief medical officer at Mirum, said in a press release. “We look forward to advancing our VANTAGE study with the goal of making volixibat available to patients living with PBC-related itch as quickly as possible.”

FDA Extends Review of Obeticholic Acid (Ocaliva) sNDA for Primary Biliary Cholangitis

In other PBC pipeline news, Intercept Pharmaceuticals announced the FDA extended its review of the supplemental New Drug Application (sNDA) for full approval of obeticholic acid (Ocaliva) for the treatment of PBC. Its action under the Prescription Drug User Fee Act (PDUFA) was expected on October 15, 2024, but was delayed with no new anticipated action date provided by the FDA. Of note, obeticholic acid is currently still available for the treatment of appropriate PBC patients in the US under accelerated approval status.

Achieving Hepatitis Goals

HBV Treatment Eligibility Greater in New WHO Guidelines Than Other International Guidelines

New research suggests that compared with the Asian Pacific Association for the Study of the Liver (APASL) 2015 guidelines, the American Association for the Study of Liver Diseases (AASLD) 2018 guidelines, and the European Association for the Study of the Liver (EASL) 2017 guidelines, the World Health Organization (WHO) 2024 hepatitis B guidelines have the highest treatment-eligible rate.

New Research Supports Universal HCV Screening Over Risk-Based Testing

Findings from this study call attention to a notable hepatitis C virus (HCV) burden among people without traditional risk factors like injection drug use, supporting the concept of universal HCV testing rather than risk-based screening. Leveraging 2013-March 2020 National Health and Nutrition Examination Survey (NHANES) data, the study estimated that 1.4 million people 12-59 years of age have current HCV infection, with more than 500,000 people in this population reporting no history of injection drug use.

Point-of-Care HCV RNA Test Could Expedite Diagnosis, Treatment in High-Risk Settings

A point-of-care HCV RNA assay (Xpert HCV) could be useful in a nontraditional, nonclinical setting for facilitating more rapid diagnosis and linkage to care among individuals under active community supervision, according to findings from this study.

In the study, the availability of a point-of-care HCV RNA assay for clinical use among individuals under active probation or parole would have enabled 30 people, including 2 with acute HCV infection, to receive an immediate HCV diagnosis.

HCPLive at ACG 2024

Patients with Liver Disease Rarely Get Alcohol Use Disorder Pharmacotherapy

An analysis of commercially insured patient data in the US found available alcohol use disorder pharmacotherapy is rarely utilized in patients with high-risk clinical history, including alcohol-associated liver disease (ALD). Findings suggest key pharmaceutical options to treat the severity and risk of mortality in patients with alcohol use disorder are disproportionately not reaching patients with ALD or hepatic decompensation, additionally highlighting a greater likelihood of receiving alcohol use disorder pharmacotherapy among patients with high-index comorbidities, psychiatric conditions, and those at older age.

Seladelpar Efficacious for Patients with PBC and Compensated Cirrhosis

Interim, long-term data from the phase 3 ASSURE trial highlight significantly improved measures of cholestasis and liver injury among patients with PBC compensated cirrhosis treated with seladelpar (Livdelzi).

Marlyn Mayo, MD: Elafibranor’s Safety, Efficacy in Patients with Advanced PBC

Findings from a new analysis of the phase 3 ELATIVE trial supporting elafibranor (Iqirvo)’s recent FDA approval for PBC suggest the peroxisome proliferator-activated receptor (PPAR) agonist’s benefit for patients with advanced disease. Results showed elafibranor provided a consistent treatment effect regardless of disease stage and had a positive impact on disease stabilization.