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
A Study of CS0159 in Patients With PBC With Inadequate Response or Intolerance to UDCA
A Randomized, Double-Blind, Placebo-controlled, Phase III Study to Evaluate the Efficacy and Safety of CS0159 in Patients with Primary Biliary Cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid (UDCA).
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.