What the trial was testing
The RESPONSE enrolled 193 patients with primary biliary cholangitis. The study was sponsored by Gilead Sciences and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
62% had a biochemical response on seladelpar vs. 20% on the comparison pill.
New England Journal of Medicine · 2024 · NCT04620733
These findings — that biochemical response at 12 months on seladelpar plus ursodeoxycholic acid — were published in the New England Journal of Medicine and represent the headline result of the study.
Researchers tracked outcomes across 193 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with primary biliary cholangitis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Seladelpar (Livdelzi) is FDA-approved and available now for primary biliary cholangitis when ursodeoxycholic acid is not enough. It also reduced the moderate-to-severe itching that drives many PBC patients up the wall. Ask a hepatologist about adding it to your existing regimen.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open primary biliary cholangitis trials
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period \[LMP\]). Information will be collected from participants, their healthcare providers, published studies, and safety databases. Reports of pregnancy linked to elafibranor from clinical trials, spontaneous reports, or literature will also be included, with steps taken to avoid duplicates. The study begins once the first participant is enrolled and ends after the last mother and child data are collected. It is planned to run for about 10 years, with infant follow-up lasting up to 2 years, for a maximum total duration of 12 years and 9 months. The program is strictly observational. All medical care, visit schedules, and treatment decisions remain with healthcare providers. Only routine medical record data will be collected, and no extra tests or procedures are required. Participation is voluntary, and written informed consent will be obtained before enrollment.
A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment
This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting. PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged. The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms including pruritus (itching) and fatigue. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. In this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 60 months (approximately 5 years).