Sanofi's Multiple Myeloma Drug Candidate SAR446523 Receives US Orphan Drug Designation
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Sanofi's investigational antibody SAR446523. The designation is for the potential treatment of relapsed or refractory multiple myeloma.
Paris, July 30, 2025 – Sanofi announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its investigational antibody, SAR446523. The drug candidate is being developed for the potential treatment of patients with relapsed or refractory multiple myeloma (R/R MM), a cancer of plasma cells.
Orphan drug designation is awarded to investigational therapies intended to treat rare medical conditions affecting fewer than 200,000 people in the United States. SAR446523 is an IgG1-based antibody designed to target G-protein coupled receptor family C group 5 member D (GPRC5D), a protein highly expressed on multiple myeloma cells with low expression in healthy tissues.
Sanofi is currently evaluating the safety and efficacy of SAR446523 in an ongoing Phase 1, first-in-human study for patients with R/R MM. The company noted that multiple myeloma remains an incurable disease requiring new therapeutic options, especially for patients who no longer respond to existing treatments.
Multiple myeloma is the second most common hematologic malignancy worldwide. Despite advancements in treatment, the disease has a high relapse rate, underscoring the need for innovative therapies.