FOSTER CITY, Calif : Gilead Sciences recently announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Livdelzi (seladelpar) 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. Doctors do not recommend Livdelzi 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 the study, 62% of participants taking Livdelzi achieved the primary endpoint of composite biochemical response at month 12, versus 20% of participants taking placebo. Treatment with Livdelzi led to normalization of alkaline phosphatase (ALP) values, a cholestatic marker that is a predictor of risk for liver transplant and death, in 25% of trial participants at month 12. None of the trial participants receiving a placebo showed this change. At month 6, Livdelzi treatment significantly reduced pruritus in patients compared with those on placebo, achieving a key secondary endpoint.
Moreover, The FDA approved Livdelzi under accelerated approval based on a reduction of ALP. The data have not demonstrated an improvement in survival or prevention of liver decompensation events. Continued approval of Livdelzi for the approved indication may be contingent on verification and description of clinical benefit in confirmatory trial(s).
“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, 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.” said Carol Roberts, President, The PBCers Organization.
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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.
However, Livdelzi, an oral PPAR delta agonist (or delpar), aims to challenge the current PBC standard of care, which often fails many people by providing an inadequate response, leaving them at risk for continued liver damage. Furthermore, Livdelzi has demonstrated a sustained efficacy and safety profile across its robust development program to date, including a capacity to normalize ALP levels for some of the people studied with PBC. Recognizing ALP levels as an important surrogate marker of disease progression in PBC, providers are shifting their focus to normalizing ALP as a treatment goal.
“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, 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.” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences.
