FDA grants ODD to Faron Pharmaceuticals’ bexmarilimab for AML treatment
Faron Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to bexmarilimab for the treatment of acute myeloid leukaemia (AML). The ODD will offer clinical development and commercialisation benefits that will ultimately strengthen the drug programme.
The drug is currently in a phase 1/2 study where it is being tested for the treatment of aggressive haematological malignancies of refractory AML and myelodysplastic syndromes (MDS) – it works by targeting myeloid cell function and activating the immune system. Phase 2 initiation is expected to begin towards the end of 2023, along with the completion of dose escalation.
The FDA’s Office of Orphan Drug Products supports the development of treatments for rare disorders that affect fewer than 200,000 people in the US. With the ODD, Faron now has exemption of FDA application fees, market exclusivity upon regulatory approval and tax credits for qualified clinical trials.
Dr Marie-Louise Fjällskog, Faron’s chief medical officer, commented: "ReceivingODD from the FDA signifies our continued progress and commitment to develop bexmarilimab as a potential treatment for AML. 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."