FDA Grants Orphan Drug Designation for MDS to Investigational CLEVER-1-Targeting Immunotherapy

The FDA has granted Orphan Drug Designation (ODD) to bexmarilimab for myelodysplastic syndromes (MDS). Bexmarilimab is an investigational immunotherapy in development by Faron Pharmaceuticals Ltd., which issued a company announcement of the FDA’s decision.

“Receiving FDA’s orphan drug designation for bexmarilimab for the treatment of myelodysplastic syndrome marks a significant milestone for Faron Pharmaceuticals as we continue to develop bexmarilimab for MDS and other cancers,” Faron chief medical officer Petri Bono, MD, PhD, commented.

A novel humanized antibody, bexmarilimab targets and binds to the CLEVER-1 immunosuppressive receptor on macrophages. This brings alteration of myeloid cell functions and recalibrates the immune system to attack hematological and solid tumors. It also increases the susceptibility of cancer cells to standard of care treatments.

The FDA already granted bexmarilimab fast track designation status and, in August 2023, an ODD for the treatment of acute myeloid leukemia (AML). The BEXMAB open-label phase 1/2 clinical trial evaluates this agent in combination with standard-of-care azacitidine for AML and MDS. Faron expects in April 2025 to report top-line efficacy results in MDS, in both frontline care and against hypomethylating-agent-refractory disease.

Reference

Inside information: FDA grants Orphan Drug Designation for bexmarilimab in MDS. News release. Faron Pharmaceuticals Ltd. March 3, 2025. Accessed April 1, 2025. https://faron.com/releases-and-publications/inside-information-fda-grants-orphan-drug-designation-for-bexmarilimab-in-mds/