Akeso’s Ligufalimab Receives FDA Orphan Drug Designation for Acute Myeloid Leukaemia (AML)
Tuesday, September 16, 2025
Akeso has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its humanised IgG4 monoclonal antibody ligufalimab (AK117), developed to target CD47 in the treatment of acute myeloid leukaemia (AML).
AML is the most common form of acute leukaemia in adults and is characterised by the rapid growth of abnormal myeloid cells in the bone marrow, blood and other tissues. Current treatment approaches, as outlined by the NCCN Guidelines®, depend on whether patients are able to undergo intensive chemotherapy. For those who cannot, options remain limited.
Ligufalimab is designed to block CD47, a protein that allows tumour cells to avoid immune attack, by preventing its interaction with the SIRPα receptor. This restores macrophage activity, enabling the immune system to better target cancer cells. Unlike other CD47-targeting agents, ligufalimab avoids red blood cell agglutination and has shown improved safety and efficacy.
Akeso is progressing the global clinical development of ligufalimab across both blood cancers and solid tumours. Patient enrolment has been completed for a randomised, double-blind Phase II trial investigating ligufalimab with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS).
The antibody has also advanced into registrational Phase III studies, making it the first CD47 monoclonal antibody to do so in solid tumours. These studies include the combination of ligufalimab and ivonescimab for first-line treatment of PD-L1-positive head and neck squamous cell carcinoma (HNSCC) and for pancreatic cancer.
Laboratory studies suggest ligufalimab works synergistically with azacitidine or venetoclax by enhancing “eat me” signals on cancer cells, leading to stronger immune responses. Clinical data have shown that the combination of ligufalimab and azacitidine is well tolerated and provides encouraging results in first-line AML treatment, with remission rates exceeding 50%.
Building on these findings, Akeso has initiated a Phase II trial to assess ligufalimab in combination with venetoclax and azacitidine for newly diagnosed AML patients unsuitable for intensive chemotherapy.
Source: prnewswire.com
