Pharmaceutical Business review

FDA grants orphan drug designation to Faron’s bexmarilimab

Bexmarilimab is intended for the treatment of acute myeloid leukemia. Credit: Animalculist/commons.wikimedia.org.

The designation will provide certain benefits to Faron including exemption of FDA application fees, tax credits for qualified trials, and on receiving regulatory approval, will get market exclusivity.

Faron Pharmaceuticals chief medical officer Dr Marie-Louise Fjallskog said: “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 being assessed in Phase I/II BEXMAB study along with standard of care to treat aggressive haematological malignancies of relapsed/refractory AML and myelodysplastic syndromes.

Nearly 181 participants aged 18 years and above were enrolled into the study.

Phase I portion is designed to identify the recommended Phase 2 dose of bexmarilimab while Phase II portion will investigate the preliminary efficacy of the combined therapy in selected indications from Phase I.

Objective responses were observed in three of five patients in the 6 mg/kg bexmarilimab + azacitidine doublet cohort, as demonstrated in the Phase I portion of the trial.

Eight out of 15 patients also showed objective responses in all three doublet dosing cohorts with one patient remaining on treatment for 13 months.

The company intends to complete the dose escalation, readout of enrichment cohorts and commence the Phase II portion of the BEXMAB trial in the fourth quarter of this year.