The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Faron Pharmaceuticals’ immunotherapy bexmarilimab, for the treatment of acute myeloid leukemia (AML).
FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. ODD provides Faron with certain benefits, such as market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials.
“Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop bexmarilimab as a potential treatment for AML,” said Chief Medical Officer Dr. Marie-Louise Fjällskog. “The designation represents a milestone in our development journey, one that we believe when combined with standard of care, will lead to better patient outcomes and improved quality of life.”
Bexmarilimab is currently in Phase I/II BEXMAB study in combination with standard of care (SoC) for the treatment of aggressive hematological malignancies of relapsed/refractory AML and myelodysplastic syndromes (MDS). Last month, Faron reported updated, positive data from the Phase I portion of the trial. Three of five patients in the 6 mg/kg bexmarilimab + azacitidine doublet cohort achieved objective responses and eight (out of 15 patients) objective responses were observed in all three doublet dosing cohorts, with one patient remaining on treatment for 13 months.
The completion of dose escalation, readout of enrichment cohorts and initiation of the Phase II portion of the BEXMAB trial are expected in Q4 2023.