The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SAR446523, an IgG1-based Antibody-Dependent Cellular Cytotoxicity (ADCC)-enhanced monoclonal antibody targeting GPRC5D, for the potential treatment of patients with relapsed or refractory multiple myeloma.

GPRC5D is highly expressed on plasma cells in MM patients, with low expression in healthy tissues. The designation is given to therapies addressing rare diseases affecting fewer than 200,000 people in the US.

“The orphan drug designation is a significant milestone in our efforts to develop innovative treatments in multiple myeloma,” said Alyssa Johnsen, MD, PhD, Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi.

The safety and efficacy of SAR446523 have not been evaluated by any regulatory authority and remain under investigation.