FDA Grants Orphan Drug Designation to Bexmarilimab for AML
31 August 2023
The US Food and Drug Administration (FDA) has given Orphan Drug Designation (ODD) to Faron Pharmaceuticals' bexmarilimab, a treatment designed for acute myeloid leukaemia (AML). This breakthrough in drug designation is set to bring advantages in clinical development, enhancing the drug's potential and paving the way for a stronger overall programme.
Bexmarilimab is currently undergoing rigorous tests. The phase 1/2 BEXMAB study is keenly assessing the combination of bexmarilimab with the standard of care for the treatment of aggressive hematological malignancies. This includes treatment avenues for relapsed/refractory AML and myelodysplastic syndromes (MDS). Recent findings from phase 1 are optimistic, showing a notable positive response in patients. Particularly notable was the success with those patients who had previously not responded to standard hypomethylating agents.
Dr Marie-Louise Fjällskog, Faron’s Chief Medical Officer, shared her insightsn a press release: “Receiving orphan drug designation 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.”
The ODD status awarded by the FDA is instrumental in promoting the research and development of drugs for rare diseases. In the US, these are disorders affecting fewer than 200,000 individuals. Benefits accompanying the ODD for Faron include an exemption from FDA application fees, guaranteed market exclusivity upon the drug’s regulatory approval, and tax benefits for any qualified clinical trials. Looking ahead, the next phases of the study are poised to take place towards the end of 2023. The completion of the dose-escalation phase, readout of enrichment cohorts, and the initiation of phase 2 are all on the horizon.
Abstract of the research
A study to assess safety, tolerability and preliminary efficacy of bexmarilimab in combination with standard of care in patients with hematological malignancies
Brief Summary: This is a study to assess the safety of increasing dose levels of bexmarilimab when combined with standard of care (SoC) in patients with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) or acute myeloid leukemia (AML); Phase 1 aims to identify the recommended phase 2 dose (RP2D) of bexmarilimab based on safety, tolerability and pharmacological activity; Phase 2 will investigate the preliminary efficacy of the combination treatment in selected indications from Phase 1. Detailed Description: This is a multicenter Phase 1/2 open-label, study to assess the safety, tolerability and preliminary efficacy of increasing doses of bexmarilimab (FP-1305) in patients with intermediate, high or very high-risk MDS, CMML with 10-19 % marrow blasts, CMML/MDS with failure to hypomethylating agent (HMA), or in patients with newly diagnosed AML non-fit for induction therapy or relapsed/refractory AML. The Phase 1 part of the study will identify a safe and tolerable bexmarilimab dose amongst four predefined dose levels using a bayesian optimal interval (BOIN) dose escalation design to identify the maximum tolerated dose (MTD) of bexmarilimab when administered in combination with SoC.
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