The drug candidate developed by the Tübingen-based biotechnology company is a selective inhibitor of MKK4, a central regulator of liver function after surgical intervention. The designation relates to the prevention of post-hepatectomy liver failure, a serious complication that can occur after extensive liver resections and that considerably impairs patients’ recovery.
The decision follows the completion of the first part of an ongoing Phase Ib/IIa trial, in which darizmetinib was well tolerated and showed favourable pharmacokinetic properties in patients undergoing minor liver resections. The next part of the trial will investigate the safety of the compound in patients undergoing major liver resections.
The US Food and Drug Administration grants orphan drug designation to medicinal products intended to treat rare diseases. The designation is linked to a range of regulatory incentives, including development support, financial benefits for clinical trials and seven years of market exclusivity following potential approval.
HepaRegeniX develops novel therapies for the treatment of acute and chronic liver diseases. The therapeutic concept was discovered by Prof. Zender, co-founder of the company, during his work at the Helmholtz Centre for Infection Research and Hannover Medical School, and was protected by patents held by both institutions. Ascenion, technology transfer partner of both institutions, has supported the project from the outset.
Further Information: HepaRegeniX's Press Release
