Zydus today received permission from the US Food and Drug Administration (FDA) to initiate the phase-II(b)/III prospective, multi-centre, randomised, double-blind, placebo-controlled adaptive clinical trial to evaluate the efficacy and safety of Saroglitazar Magnesium in subjects with Primary Biliary Cholangitis (PBC), the company said in a statement.

It further mentioned that The EPICS-IIITM trial will randomise 192 subjects in a 1:1:1 ratio to Saroglitazar 1 mg, Saroglitazar 2 mg or placebo and will study the Saroglitazar Magnesium (1 or 2 mg) relative to placebo based on the composite endpoints of Alkaline Phosphatase (ALP), total bilirubin, Liver Stiffness Measurement (LSM) by FibroScan, liver enzyme parameters (ALT, AST, GGT, total bilirubin and albumin), lipid parameters (TG, LDL-C, HDL-C, VLDL-C, total cholesterol and non-HDL-C), health-related quality of life using PBC 40 questionnaire (a patient-derived, disease-specific quality of life measure developed and validated for use in PBC) and other outcome measures over a period of 52 weeks. The change in pruritus (60- 70 per cent of patients with PBC liver disease develop systemic itch of the skin that significantly impairs activity and sleep) will be studied using 5-Domains (5-D) itch scale, patient global impression of change scale, patient global therapeutic benefit scale and patient global impression of worst itch severity scale. The EPICS-IIITM phase-II(b)/III trial will be led by Professor Naga Chalasani, MD, Interim Chair, Department of Medicine, Indiana University School of Medicine as Principal Investigator.

Speaking on the new development, Pankaj R Patel, Chairman, Cadila Healthcare, said, “Primary Biliary Cholangitis (PBC) is a severe liver disease leading to cirrhosis of the liver, liver failure, and possibly death. PBC patients have limited treatment options and approximately 50 per cent of the PBC patients are not able to benefit from the existing therapy due to lack of response or intolerable side effects. Based on the positive results from our earlier phase-II study, we have now initiated the pivotal EPICS III trial in both cirrhotic and non-cirrhotic PBC patients.”

The phase-II (a) EPICS trials results published in the Journal of Hepatology has demonstrated that
Saroglitazar holds immense potential based on its safety and efficacy profile. The global market for PBC treatment is expected to grow at a CAGR of 36.3 per cent from 2018-2026, and is expected to reach $10.8 billion by 2026, as per Coherent market insights, the statement added.

It also notified that Saroglitazar Mg is an investigational compound in the USA, and is yet to be approved by the US FDA or European Medicines Agency (EMA). The US FDA has granted ‘Orphan Drug Designation’ and ‘Fast Track Designation’ to SaroglitazarMg for PBC. The EMA has designated “Saroglitazar Magnesium” with Orphan status for Treatment of Primary Biliary Cholangitis. Orphan drug designation provides eligibility for certain development incentives, regulatory fee exemptions, seven-year marketing exclusivity upon the US FDA approval and a 10-year period of market exclusivity if the treatment eventually is approved by EMA.