FDA Prioritizes Review of Zydus’ Saroglitazar for Rare Liver Disease

Zydus Therapeutics has received US Food and Drug Administration (FDA) Priority Review for its New Drug Application (NDA) seeking approval of saroglitazar in primary biliary cholangitis (PBC). The agency has set a Prescription Drug User Fee Act (PDUFA) target action date of November 27, 2026.

Priority Review shortens the FDA’s evaluation timeline from the standard 10 months to approximately 6 months. If approved, Zydus anticipates a US launch in the fourth quarter of fiscal year 2027.

Addressing an Unmet Need

PBC is a rare autoimmune liver disease that progressively damages bile ducts, leading to fibrosis, cirrhosis, liver transplantation, or death. While ursodeoxycholic acid (UDCA) remains the standard first-line treatment, many patients continue to experience disease progression or cannot tolerate therapy. The NDA covers saroglitazar in combination with UDCA for adults with an inadequate response to standard treatment and as monotherapy for patients unable to tolerate UDCA.

Phase 3 EPICS‑III Results

The Priority Review is supported by data from the EPICS-III Phase 3 trial (NCT05133336), a randomized, double-blind, placebo-controlled study involving 148 patients with an inadequate response to or intolerance of UDCA.

At Week 52, saroglitazar met the primary endpoint, with 56.7% of treated patients achieving biochemical response compared with 9.8% of patients receiving placebo (p<0.001). The endpoint required ALP below 1.67 times the upper limit of normal, at least a 15% reduction from baseline, and normal bilirubin levels.

Among patients with baseline ALP levels at or below three times the upper limit of normal, biochemical response reached 83.1% with saroglitazar versus 14.7% with placebo.

Saroglitazar also reduced ALP by 33.5% from baseline, while ALP increased by 6.5% in the placebo group, resulting in a treatment difference of 40.1%. ALP is an established surrogate marker associated with long-term outcomes in PBC.

The therapy significantly improved pruritus at Week 24, although differences between treatment groups were not statistically significant at Week 52.

Safety Profile

Saroglitazar was generally well tolerated. Most treatment-emergent adverse events were mild to moderate. Serious adverse events occurred in 6.3% of patients receiving saroglitazar compared with 11.1% of those receiving placebo. Investigators reported no treatment-related deaths.

Mechanism of Action

Saroglitazar is an investigational dual peroxisome proliferator-activated receptor (PPAR) α/γ agonist that targets bile acid toxicity and liver inflammation, two key drivers of PBC progression. This mechanism differentiates it from other investigational therapies such as seladelpar (PPAR‑δ) and elafibranor (PPAR α/δ), potentially offering a broader impact on disease pathways. The therapy has received FDA Orphan Drug Designation, Fast Track Designation, and Priority Review status.

Implications for Patient Care

Detailed EPICS‑III findings will be presented during a late‑breaking session at the European Association for the Study of the Liver (EASL) Congress 2026 in Barcelona. Zydus is preparing medical affairs and commercialization activities ahead of potential approval.

If approved, saroglitazar would expand treatment options for patients whose disease remains inadequately controlled on current therapy and mark Zydus’ entry into the US rare liver disease market.

With its differentiated dual PPAR α/γ mechanism, the therapy would join a competitive second‑line PBC landscape and potentially offer a novel approach to addressing bile acid toxicity and inflammation in this underserved patient population.

Reference

Microsoft Word – Press Release_Zydus Therapeutics NDA for Saroglitazar to treat (PBC) granted priority review by the US FDA