GlobeNewswire·Oct 21, 2026, 4:05 PM ET

Cardiff Oncology Receives FDA Orphan Drug Designation for Onvansertib in Chronic Myelomonocytic Leukemia

The U.S. FDA granted Orphan Drug Designation to onvansertib for treating chronic myelomonocytic leukemia (CMML).
Mani Mohindru, CEO of Cardiff Oncology, stated that 40-50% of myeloproliferative CMML patients harbor RAS-pathway mutations linked to aggressive disease.
Onvansertib demonstrated preliminary efficacy in approximately 40% of myeloproliferative CMML patients in a Phase 1 dose-ranging trial.
Clinical benefits included one patient with a 100% platelet response, one with normalized white blood cells, one with a spleen response, one with optimal marrow response, and three with stable disease.
Orphan Drug Designation offers potential benefits such as tax credits, fee exemptions, & up to seven years of market exclusivity upon approval.
Cardiff Oncology plans to advance onvansertib in a registrational trial for first-line RAS-mutated metastatic colorectal cancer using a 30 mg dose combined with FOLFIRI/bevacizumab.
The drug is also being evaluated in investigator-initiated studies for metastatic pancreatic ductal adenocarcinoma, small cell lung cancer, triple-negative breast cancer, & CMML.
CMML is a rare, aggressive hematologic malignancy primarily affecting older adults with limited current treatment options.
Cardiff Oncology is a clinical-stage biotechnology company focused on developing PLK1 inhibition therapies for various cancers.