Sanofi earns orphan drug designation from USFDA for multiple myeloma drug
Paris: Sanofi has received orphan drug designation from the US Food and Drug Administration (FDA) for SAR446523, an IgG1-based Antibody-Dependent Cellular Cytotoxicity-enhanced (ADCC) monoclonal antibody (mAb) targeting G-protein coupled receptor family C group 5 member D (GPRC5D) for the potential treatment of patients with relapsed or refractory multiple myeloma (R/R MM).
GPRC5D is highly expressed on plasma cells in MM patients, with low expression in healthy tissues. The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the US.
“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 an investigational IgG1-based mAb designed to target GPRC5D, which is highly expressed on plasma cells, with an engineered fragment crystallizable domain to enhance antibody dependent cell-mediated cytotoxicity. This innovative approach aims to improve the efficacy of treatment for MM, a rare and challenging cancer of plasma cells. Subcutaneous SAR446523 is currently being evaluated in an ongoing phase 1, first-in-human study in patients with R/R MM (clinical study identifier: NCT06630806). SAR4465523 originates from Sanofi Research in Vitry-sur-Seine, France.
Multiple myeloma is considered a rare disease, yet MM is the second most common hematologic malignancy with more than 180,000 people diagnosed with MM each year, globally. Despite available treatments, MM remains an incurable malignancy with an estimated 62% five-year survival rate for newly diagnosed patients. There is a need for new frontline therapeutic options for all patients, especially for those who are transplant ineligible, due to high attrition rates in subsequent lines of therapy. Since MM does not have a cure, most patients will relapse and stop responding to therapies they have received.
The safety and efficacy of SAR446523 has not been evaluated by any regulatory authority and is still under investigation.
