Akeso Secures U.S. Orphan Drug Status for Cancer Therapy Ligufalimab

Chinese biotech company Akeso Inc. (HK:9926) announced that its experimental cancer drug ligufalimab (AK117) has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).

The ODD program is designed to encourage the development of therapies targeting rare diseases. Benefits include tax credits, waived fees, and up to seven years of U.S. market exclusivity once the drug gains approval. According to Akeso, this milestone strengthens its global oncology pipeline and supports ongoing research into therapies for blood cancers and solid tumors.

Ligufalimab is a monoclonal antibody that targets CD47, a protein often called the “don’t eat me” signal that allows tumor cells to evade immune detection. By blocking CD47, the therapy aims to enhance the body’s ability to fight cancer. The drug is currently being evaluated in several clinical trials, including Phase III studies in pancreatic cancer and head and neck squamous cell carcinoma.

In addition, Akeso is advancing combination therapy studies for AML patients who are not eligible for intensive chemotherapy, a group with limited treatment options. The company emphasized that this designation could accelerate development timelines while improving treatment accessibility for high-need patient populations.

Following the announcement, Akeso’s Hong Kong-listed shares rose nearly 1% to HK$130.10 as of 04:47 GMT, reflecting investor optimism over the FDA’s decision.

With the ODD status, Akeso moves closer to positioning ligufalimab as a potential breakthrough therapy in both hematologic and solid tumor cancers. The company’s progress highlights the growing role of Chinese biotech firms in the global oncology landscape, particularly in developing innovative immuno-oncology treatments.