Sanofi announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to SAR446523, an investigational monoclonal antibody designed to target GPRC5D for the potential treatment of patients with relapsed or refractory multiple myeloma. The FDA’s orphan drug designation is given to therapies addressing rare diseases or conditions affecting fewer than 200,000 people in the United States.
SAR446523 is an IgG1-based antibody engineered to enhance antibody-dependent cell-mediated cytotoxicity. It targets GPRC5D, a protein highly expressed on plasma cells in multiple myeloma patients but found at low levels in healthy tissues. According to Sanofi, the safety and efficacy of SAR446523 have not yet been evaluated by any regulatory authority and remain under investigation.
“The orphan drug designation is a significant milestone in our ongoing efforts to develop innovative treatments in multiple myeloma,” said Alyssa Johnsen, MD, PhD, Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi. “This underscores our commitment to multiple myeloma, a disease for which we have acquired strong expertise with the development of another widely used and approved immunotherapy treatment.”
SAR446523 is currently being tested in an ongoing phase 1 clinical study involving patients with relapsed or refractory multiple myeloma (clinical study identifier: NCT06630806). The compound was developed by Sanofi Research in Vitry-sur-Seine, France.
Multiple myeloma remains incurable despite existing therapies. Each year more than 180,000 people worldwide are diagnosed with this rare cancer of plasma cells. In the United States, newly diagnosed patients have an estimated five-year survival rate of about 62%. There is a continued need for new therapeutic options as most patients eventually relapse and become resistant to available treatments.
Sanofi states its long-term commitment to oncology research includes developing first- and best-in-class immunological and targeted therapies for cancers that are difficult to treat or have high unmet needs.
For more information on clinical studies related to multiple myeloma, visit www.clinicaltrials.gov.
Sanofi describes itself as an R&D-driven biopharmaceutical company focused on inventing medicines and vaccines that address urgent healthcare challenges globally. The company is listed on EURONEXT: SAN and NASDAQ: SNY.
