HepaRegeniX has received U.S. FDA Orphan Drug Designation for darizmetinib for the prevention of post‑hepatectomy liver failure. The therapy is a selective inhibitor of MKK4 designed to accelerate and enhance the liver’s natural regenerative capacity in healthy and diseased tissue.
Post‑hepatectomy liver failure is a serious complication that can limit a patient’s ability to recover after extensive liver resection. Linda Greenbaum, Chief Medical Officer of HepaRegeniX, said: “Post-hepatectomy liver failure is a serious and potentially life-threatening complication of liver resection and can limit the ability of patients to recover following extensive resection.”
She added: “Darizmetinib is designed to enhance the liver’s regenerative capacity and support recovery when the remaining liver may not have sufficient capacity to regenerate and maintain essential function following surgery.”
Elias Papatheodorou, Chief Executive Officer of HepaRegeniX, explained: “Receiving the Orphan Drug Designation for darizmetinib is an important regulatory milestone for HepaRegeniX and supports its continued development for patients undergoing liver resection.”
He added: “As we advance darizmetinib into later-stage clinical development, the designation strengthens our ongoing engagement with the FDA and advances our goal of bringing a new treatment option to patients.”
The designation follows completion of the first pilot part of the phase Ib/IIa study, which showed favourable tolerability and pharmacokinetic profiles in patients undergoing minor liver resection. The second pilot part is expected to begin in the third quarter of 2026 and will assess safety and pharmacokinetics in patients undergoing major liver resection.
The FDA grants Orphan Drug Designation to therapies intended for rare diseases affecting fewer than 200,000 people in the U.S. Sponsors qualify for incentives including tax credits for clinical trials, exemption from user fees and regulatory guidance throughout development.
The approved product will receive seven years of post‑approval market exclusivity in the U.S. for the designated condition.
