India Pharma Outlook Team | Monday, 04 August 2025
The U.S. Food and Drug Administration (FDA) have made Orphan Drug Designation (ODD) of SAR446523, a GPRC5D monoclonal antibody, which is currently in development by Sanofi as a potential therapy for relapsed or refractory multiple myeloma.
SAR446523 is an IgG1-based antibody that is developed for antibody-dependent cellular cytotoxicity (ADCC), to help harness the immune system to kill cancer cells.
Multiple myeloma, an extremely rare and highly aggressive blood cancer, falls under the FDA's orphan drug criteria as it impacts less than 200,000 individuals in the U.S.
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SAR446523 targets GPRC5D, the receptor that is expressed at a much higher level on plasma cells in patients with multiple myeloma, and is present at negligible levels in healthy tissues, making it ideal for targeted immunotherapy.
“This orphan drug designation is a significant milestone in our commitment to advancing innovative therapies in multiple myeloma,” said Alyssa Johnsen, MD, PhD, Global Therapeutic Area Head of Immunology and Oncology Development at Sanofi.
Orphan Drug Designation is designed to accelerate the commercialization of drugs intended for rare conditions, which includes benefits like market exclusivity, tax credits, and fee waivers. While the safety and efficacy of SAR446523 have not yet been evaluated by any regulatory agency, early development marks a critical step in expanding treatment options for patients facing limited alternatives.
As research continues, SAR446523 holds potential to become a targeted therapy that addresses a major unmet need in the multiple myeloma treatment landscape.
