FDA Grants Accelerated Approval to Gilead Treatment for Primary Biliary Cholangitis

Foster City, Ca.-based Gilead Sciences announced in a press release on Aug. 14, 2024 that its Livdelzi (seladelpar), a 10-mg oral treatment for primary biliary cholangitis (PBC), was given an accelerated approval by FDA for use in patients with PBC, either in combination with ursodeoxycholic acid (UDCA) for adults who have an inadequate response to UDCA, or as monotherapy in those who cannot tolerate UDCA (1,2).

Although PBC, which primarily affects women, is relatively rare, occurring in 1 in 1000 women over the age of 40 in the United States or about 130,000 people in total, it is a chronic inflammatory liver disease, the progression of which can present an increased risk of liver-related mortality (1). It is characterized by cholestasis, or impaired bile flow, and the accumulation of toxic bile acids in the liver, which combined lead to inflammation and eventual destruction of the bile ducts, as well as increased levels of three enzymes found primarily in the liver (alkaline phosphatase [ALP], alanine transaminase, and gamma-glutamyl transferase) and total bilirubin.

“More people are being diagnosed with PBC, impacting people of varied ages, gender, race and ethnicity. Those living with PBC share common symptoms, including incessant itching or skin-crawling sensations, as well as debilitating fatigue that is made worse by the itching at night,” Carol Roberts, president of The PBCers Organization, a 501(c)(3) based in Rochester, NY, said in the press release (1,3). “The availability of a new treatment option that can help reduce this intense itching while also improving biomarkers of active liver disease is a milestone for our community.”

A Phase III study (RESPONSE) revealed that 62% of patients who were given Livdelzi achieved the primary endpoint of biochemical response as of month 12, versus 20% of study participants given placebo. Additionally, administration of Livdelzi helped to normalize ALP values in 25% of participants after 12 months. Among the enzymes commonly found in the liver, ALP is a cholestatic marker that serves as a predictor for liver transplant and death. The change in ALP was not observed in any patients in the study who received placebo (1).

“People living with PBC have been waiting for treatment advancements for many years. Today’s approval of Livdelzi, with its distinct profile, provides them with an important new option,” said Daniel O’Day, chairman and chief executive officer of Gilead Sciences, also in the press release. “We look forward to leveraging Gilead’s long-standing expertise in liver disease to bring this promising new treatment to all those who could benefit.”

Despite the accelerated approval for the selected patient groups, Livdelzi is not recommended for people who have, or may develop, decompensated cirrhosis (1).

References

1. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.
2. Gilead Sciences. Livdelzi – Seladelpar 10mg Capsules. gilead.com/science-and-medicine/medicines (accessed Aug. 15, 2024).
3. PBCers Organization. Contact Us–PBCers.org. PBCers.org/contact-us/ (accessed Aug. 15, 2024).