The FDA granted orphan drug designation to ligufalimab for AML, reflecting its potential as an immune-based remedy that will supply new choices for sufferers.
Ligufalimab (AK117), a next-generation antibody that targets a protein on most cancers cells, has been granted orphan drug designation by the U.S. Meals and Drug Administration (FDA) for the remedy of acute myeloid leukemia (AML), in accordance with a information launch from Akesobio.
The orphan drug designation program was created by the FDA to encourage the event of therapies for uncommon ailments. Medication with this standing obtain assist from the FDA throughout growth, tax incentives and, if authorised, as much as seven years of market exclusivity. For sufferers, this implies the company sees promise in a remedy that would assist meet an unmet medical want.
How Ligufalimab Works
Ligufalimab is a sort of immunotherapy known as a monoclonal antibody. It really works by concentrating on CD47, a protein on the floor of most cancers cells. Usually, CD47 sends a “don’t eat me” sign to the immune system, permitting most cancers to cover. By blocking CD47, ligufalimab helps immune cells known as macrophages higher acknowledge and destroy most cancers cells, slowing or stopping tumor development.
In contrast to different CD47-targeting medicine, ligufalimab is designed to keep away from clumping with pink blood cells, an issue that may make remedies much less secure and fewer efficient.
Early Ends in AML
In laboratory research, ligufalimab labored particularly nicely when mixed with different medicine resembling Vidaza (azacitidine) or Venclexta (venetoclax). These mixtures made it simpler for the immune system to assault leukemia cells, which can be particularly essential for sufferers who can not obtain normal chemotherapy.
Early medical trials have additionally proven constructive outcomes. When ligufalimab was given with Vidaza as an preliminary remedy for AML, the drug was typically secure and nicely tolerated, even at increased doses. About half of sufferers achieved an entire remission, and greater than half had a powerful general response to remedy.
Different Ongoing Analysis
Ligufalimab can be being studied in strong tumors, making it the primary CD47-targeting antibody to succeed in giant, late-stage part 3 trials on this setting. Two research are ongoing: one in sufferers with head and neck squamous cell carcinoma whose tumors categorical PD-L1, and one other in pancreatic most cancers.
Why New Therapies Are Wanted in AML
Remedy choices for AML stay restricted, particularly for older sufferers or those that can not tolerate intensive chemotherapy. The FDA has authorised mixtures of Venclexta with medicine resembling Vidaza, Dacogen (decitabine), or low-dose cytarabine for these sufferers. Whereas these regimens have prolonged survival for some, greater than half relapse inside months, and common survival stays about one 12 months.
This highlights the necessity for brand spanking new approaches, particularly ones like ligufalimab that use the immune system another way.
What Is AML?
AML is the most typical kind of acute leukemia in adults. It develops when immature white blood cells, known as myeloid blasts, develop uncontrollably within the bone marrow and blood. These irregular cells crowd out wholesome ones and should unfold exterior the bone marrow. Remedy usually will depend on a affected person’s general well being. Some can obtain intensive chemotherapy, whereas others might have much less aggressive approaches. For sufferers who can not tolerate robust remedies, new therapies beneath research supply hope.
Wanting Forward
Past AML, ligufalimab can be being evaluated in sufferers with higher-risk myelodysplastic syndromes (HR-MDS). A part 2 trial testing the drug together with Vidaza has accomplished enrollment. The research is rigorously designed in order that neither sufferers nor docs know who’s receiving which remedy, serving to guarantee outcomes are dependable.
With its new orphan drug designation and inspiring early information, ligufalimab represents a possible new choice within the pipeline for sufferers with AML who urgently want higher therapies.
Reference
- “Akeso’s Ligufalimab (CD47 mAb) Receives FDA Orphan Drug Designation for Acute Myeloid Leukemia (AML).” Akesobio. Information Launch. Sep. 16, 2025.
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