Gilead Sciences has received the US Food and Drug Administration (FDA) approval for Livdelzi (seladelpar) as a second-line treatment for people with primary biliary cholangitis (PBC).
Livdelzi is an oral, peroxisome proliferator activated receptor (PPAR) delta agonist (delpar), with the potential to challenge the current PBC standard of care.
The drug has shown a sustained efficacy and safety profile across its development programme and helped normalise ALP levels for some of the study participants with PBC.
Livdelzi is indicated for the treatment of PBC, in combination with ursodeoxycholic acid (UDCA) in adults who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
The US FDA approved the drug under its accelerated approval pathway, based on its ability to reduce the levels of alkaline phosphatase (ALP).
The US biotechnology company said that continued approval for this indication is expected based on verification and description of clinical benefit in confirmatory trials.
The PBCers Organisation president Carol Roberts said: “More people are being diagnosed with PBC, impacting people of varied age, 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.”
The FDA granted accelerated approval primarily based on data from the placebo-controlled Phase 3 RESPONSE study of Livdelzi as monotherapy or in combination with UDCA.
In the Phase 3 study, 62% of participants treated with Livdelzi achieved the primary endpoint of composite biochemical response at month 12, compared to 20% with placebo.
A 25% of participants treated with Livdelzi experienced the normalisation of ALP, a cholestatic marker that is a predictor of risk for liver transplant and death.
In addition, treatment with Livdelzi also helped reduce pruritus (chronic itch), a common symptom that significantly worsens the quality of life in people with PBC.
Gilead said that its Livdelzi showed statistically significant and durable improvements in both pruritus and cholestasis markers related to the risk of disease progression.
The most common adverse events in the study include headache, abdominal pain, nausea, abdominal distension (swelling) and dizziness, with no serious adverse events reported.
Gilead Sciences chairman and CEO Daniel O’Day said: “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.”
