FDA Grants Accelerated Approval for Seladelpar to Treat Primary Biliary Cholangitis

The US Food and Drug Administration (FDA) has granted accelerated approval for seladelpar (brand name Livdelzi) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

“The FDA approval of seladelpar is exciting news for PBC patients and their physicians,” commented Alan Bonder, MD, associate professor of medicine at Harvard and medical director of liver transplantation at Beth Israel Deaconess Medical Center in Boston, Massachusetts. “This marks a significant advancement in therapy options. It's incredible to see the progress being made in the treatment of this rare disease.”

PBC is a rare, chronic, autoimmune disease of the bile ducts that affects approximately 130,000 Americans, primarily women, and can cause liver damage and possible liver failure if untreated. The disease currently has no cure.

The FDA approved seladelpar under accelerated approval based on a reduction of alkaline phosphatase (ALP), an important surrogate marker of disease progression and risk of liver transplantation and death in PBC.

Seladelpar is an oral, peroxisome proliferator activated receptor (PPAR) delta agonist, or delpar, that has demonstrated a sustained efficacy and safety profile across its development program to date. The drug has not demonstrated improvement in survival or prevention of liver decompensation events to date. The continued approval of seladelpar for PBC may be contingent on verification and description of clinical benefit in confirmatory trial(s).

Seladelpar is not recommended for people who have or develop decompensated cirrhosis.

The accelerated approval was based primarily on data from the pivotal placebo-controlled Phase 3 RESPONSE study in which 62% of participants treated with seladelpar achieved the primary endpoint of composite biochemical response at month 12, versus 20% of participants taking placebo. One quarter of the participants treated with seladelpar achieved normalization of ALP values at month 12, while ALP normalization was not achieved by any participants in the placebo cohort. Patients treated with seladelpar also demonstrated a statistically significant reduction in pruritus at month 6 when compared with placebo.

Reference:

Gilead’s Livdelzi (seladelpar) granted accelerated approval for primary biliary cholangitis by U.S. FDA. Press release. Gilead Sciences, Inc. August 14, 2024. Accessed August 15, 2024. https://www.gilead.com/news-and-press/press-room/press-releases/2024/8/gileads-livdelzi-seladelpar-granted-accelerated-approval-for-primary-biliary-cholangitis-by-us-fda