FDA Orphan Drug Designation for AML and Pancreatic Cancer Treatment Possibly More Effective Than Chemotherapy

The FDA has just provided AB001 the Orphan Drug designation for both acute myeloid leukemia (AML) and pancreatic cancer. This small molecule was developed by Vopec Pharmaceuticals and Agastiya Biotech.


This small molecule has a dual mechanism of action. It is a PD-L1 checkpoint inhibitor, but the therapy additionally targets aberrant intracellular signaling pathways like P13K, RAS, and Wnt/beta-catenin around the tumor. The dual mechanism has already been demonstrated in animal models of various cancers. Researchers have tested non-PDL1+ cancers, liquid tumors, and solid tumors. These animal models have demonstrated the ability of this investigative therapy to do things that other treatments have not yet been able to. For instance, no other drug has been able to combat beta-catenin.

Studies have shown AB001 has many advantages compared to the standard of care treatment for pancreatic cancer called Gemcitabine. Its utility in leukemia is also shown. Preclinical models of acute lymphoblastic leukemia demonstrated that AB001 created anti-leukemia activity. Researchers suspect this is accomplished through its inhibition of the VEGFR2-mediated signaling pathways.

Chemotherapy vs. AB001

Standard chemotherapy treatments are extremely effective at treating many cancers. However, despite how well they kill cancerous tumor cells, they have very high toxicity and also harm the normal cells. This leads to devastating side effects. Patients facing chemotherapy are faced with depleted energy and overall wellbeing during treatment.

Studies have demonstrated that AB001 is able to kill cancerous tumor cells while not harming normal cells. This could be life changing for patients. Further, this treatment targets stem cells specifically, which disrupts relapse mechanisms and drug resistance.

Looking Forward

All in all, this treatment has been studied in five different cancers. It has been able to prevent cells from turning into tumors and also maintains the tumor suppressive genes in an active state. Researchers are extremely hopeful about the utility of this therapy for many different cancers. It could lead to improved quality of life and improved outcomes for many patients.

You can read more about this investigative therapy here.

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