Pharma Focus Asia

CymaBay Therapeutics Discovered Novel Seladelpar for the Treatment of Primary Biliary Cholangitis

CymaBay Therapeutics has discovered novel Seladelpar, designed to address primary biliary cholangitis (PBC) and associated pruritus in adults who do not have cirrhosis or have compensated cirrhosis classified as Child Pugh A.

Seladelpar is a potent, highly selective, orally administered PPARδ agonist, often referred to as a "delpar." Phase 3 trials have yielded compelling evidence, showcasing statistically significant enhancements in the management of PBC-related cholestatic pruritus.

Seladelpar is an investigational treatment engineered to address the needs of individuals afflicted by PBC. It is the first-in-class oral medication, functioning as a selective activator of the peroxisome proliferator-activated receptor (PPAR) delta. 

This innovative drug has demonstrated its capability to influence pivotal metabolic and liver disease pathways, particularly in medical conditions characterised by significant unmet needs. 

Both preclinical and clinical data provide strong support for its capacity to regulate genes associated with bile acid synthesis, inflammation, fibrosis, and the metabolism, storage, and transport of lipids.

Primary Biliary Cholangitis (PBC) is a rare, chronic inflammatory liver disorder predominantly affecting women, with an estimated incidence of 1 in 1,000 women over the age of 40, totalling approximately 130,000 individuals in the United States.

PBC is characterised by a condition known as cholestasis, in which there is impaired bile flow. This leads to the accumulation of harmful bile acids within the liver, resulting in inflammation and damage to the bile ducts. As a consequence, levels of alkaline phosphatase (ALP) and total bilirubin in the bloodstream tend to rise.

Common early indicators of PBC include pruritus, or persistent itching, and fatigue. As PBC progresses, it is associated with an increased risk of liver-related mortality.

The U.S. Food and Drug Administration (FDA) has granted breakthrough designation for seladelpar to now encompass its application in treating primary biliary cholangitis (PBC).


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