Zydus Cadila, an Indian pharmaceutical company, has announced the success of its late-stage trial for saroglitazar in the treatment of primary biliary cholangitis (PBC). The company is now preparing to submit a New Drug Application (NDA) to the US FDA. Saroglitazar is a novel, oral, dual peroxisome proliferator-activated receptor (PPAR) agonist that has shown promising results in reducing alkaline phosphatase (ALP) levels, a key marker of PBC.
The phase 3 trial, which enrolled 130 patients, met its primary endpoint, demonstrating a statistically significant reduction in ALP levels from baseline to week 24. The results also showed that saroglitazar was well-tolerated, with a safety profile comparable to that of the placebo group. The trial’s success marks a significant milestone for Zydus, as it seeks to bring a new treatment option to patients with PBC, a chronic and progressive liver disease with limited treatment options.
PBC is a rare autoimmune disease that affects the bile ducts in the liver, leading to inflammation, scarring, and ultimately, liver failure. Current treatments for PBC, such as ursodeoxycholic acid (UDCA), are often ineffective in reducing ALP levels and slowing disease progression. Saroglitazar’s dual PPAR agonism mechanism is thought to provide a unique benefit in treating PBC, as it targets both PPAR-alpha and PPAR-gamma receptors, which play a crucial role in lipid metabolism and inflammation.
The success of saroglitazar in the phase 3 trial has significant implications for Zydus, as it prepares to enter the US market. The company plans to submit an NDA to the FDA, which, if approved, would make saroglitazar the first dual PPAR agonist available for the treatment of PBC in the US. Zydus’s submission is expected to be based on the results of the phase 3 trial, as well as additional data from ongoing and planned clinical studies.
The approval of saroglitazar in the US would not only expand treatment options for patients with PBC but also provide a new revenue stream for Zydus. The company has already received orphan drug designation for saroglitazar in the US, which provides incentives for the development of treatments for rare diseases, including tax credits, marketing exclusivity, and reduced regulatory fees. With its late-stage success and impending US application, Zydus is poised to make a significant impact in the treatment of PBC, a disease with limited treatment options and a high unmet medical need.