Saroglitazar

The adjusted U.S. prevalence of PBC in 2021 was 40.9 per 100,000 adults, corresponding to an estimated 105,506 adults. In the ITCH-E study, activity impairment was 57% in the moderate-to-severe pruritus group versus 36% in the no or mild pruritus group. A separate observational analysis reported one-year adjusted healthcare costs of $39,949 with pruritus versus $25,695 in controls. These observational comparisons describe disease burden rather than treatment effects.

In the phase 3 portion of EPICS-III, 148 participants were randomized. The sponsor reported biochemical response at week 52 in 56.7% receiving saroglitazar and 9.8% receiving placebo. Serious adverse events were reported in 6.3% and 11.1%, respectively. The EASL Congress report described a statistically significant pruritus improvement at six months that was not maintained at 12 months. These results support a biochemical treatment effect but do not establish a reduction in liver transplantation or mortality.

The report contains an illustrative managed-access model for the Spanish National Health System, available as a preprint abstract. It assumes a net price of €25,000 per year and reports modeled net health-system costs of €4,371,005 over 24 months, with €586,495 in savings relative to its modeled price without risk sharing. These are model assumptions and outputs, not an announced commercial price or a U.S. budget-impact estimate.

Saroglitazar remains investigational for PBC in the United States. The sponsor reports Priority Review with a PDUFA target action date of November 27, 2026. The proposed population consists of adults with an inadequate response to UDCA, or patients unable to tolerate UDCA. The available phase 3 evidence primarily addresses biochemical response over 52 weeks; longer-term clinical benefit remains to be established.