US Approves Gilead's Seladelpar for PBC

On Wednesday, the FDA granted accelerated approval for Gilead Sciences' new drug, seladelpar, to treat primary biliary cholangitis (PBC). This approval applies to its use in combination with ursodeoxycholic acid (UDCA) for adults who have shown an inadequate response to UDCA, as well as for monotherapy in patients who are unable to tolerate UDCA. The drug, now named Livdelzi, was acquired by Gilead through a $4.3 billion acquisition of CymaBay earlier this year.

The approval is supported by data from the Phase III RESPONSE study, which demonstrated that 62% of patients treated with Livdelzi achieved the primary endpoint of a composite biochemical response at 12 months. This compares favorably to just 20% of patients who were given a placebo. Additionally, Livdelzi was shown to normalize alkaline phosphatase (ALP) levels—a key cholestatic marker associated with liver transplant risk and mortality—in 25% of patients by the 12-month mark. This normalization was not observed in any of the patients who received the placebo.

Furthermore, an important secondary endpoint measured the change in pruritus scores from baseline at six months. Patients treated with Livdelzi experienced a statistically significant reduction in pruritus compared to those who were given a placebo.

The development and subsequent approval of Livdelzi mark a significant advancement in the treatment options available for patients suffering from PBC, particularly for those who do not adequately respond to, or cannot tolerate, the existing standard treatment, UDCA.