FDA Grants Orphan Drug Status to Akeso’s Ligufalimab for AML

Akeso Inc. announced that its monoclonal antibody ligufalimab (AK117), targeting CD47, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration for the treatment of acute myeloid leukemia (AML).

The ODD status provides Akeso with benefits including regulatory guidance, tax incentives, and up to seven years of market exclusivity upon approval, aimed at encouraging development of therapies for rare diseases.

Ligufalimab is currently in international clinical trials for both hematologic cancers and solid tumors. In AML, it has shown promising safety and efficacy results when combined with azacitidine, achieving a 50% complete remission rate and a 55% composite complete remission rate in early studies. Akeso recently initiated a Phase II trial investigating ligufalimab with venetoclax and azacitidine for AML patients who are not candidates for intensive chemotherapy.

Beyond AML, ligufalimab is being tested in other cancers, including head and neck squamous cell carcinoma and pancreatic cancer, where it is part of Phase III registrational trials in combination with ivonescimab.

AML is a severe blood cancer with limited treatment options, especially for patients ineligible for intensive chemotherapy. Existing therapies offer short remission durations and poor survival rates, underscoring the urgent need for new treatments.

Ligufalimab works by blocking CD47, a “don’t eat me” signal that helps tumor cells evade immune destruction. By disrupting this pathway, it enhances the ability of macrophages to engulf and destroy cancer cells, offering a novel immunotherapeutic approach.

With the FDA’s orphan drug designation, Akeso advances its mission to develop innovative treatments for patients with serious cancers and significant unmet needs.