Sanofi’s Rilzabrutinib Granted Orphan Drug Designation for Rare Diseases

Sanofi (EPA: SAN, NASDAQ: SNY) announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its oral Bruton’s tyrosine kinase (BTK) inhibitor, rilzabrutinib, for the treatment of warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD). These designations highlight the potential of rilzabrutinib to address significant unmet medical needs in these rare conditions.

Disease Background

• wAIHA: A rare, life-threatening autoimmune disorder characterized by the premature destruction of red blood cells due to autoantibodies, leading to hemolysis.
• IgG4-RD: A rare, progressive, and chronic fibro-inflammatory condition that can affect multiple organs, potentially leading to irreversible organ damage and dysfunction.

Clinical Trial Results

• wAIHA: A Phase IIb study demonstrated clinically meaningful improvements in response rates and disease markers among patients treated with rilzabrutinib.
• IgG4-RD: A Phase IIa study showed that 52 weeks of treatment with rilzabrutinib reduced disease flares, lowered disease markers, and minimized glucocorticoid use, with consistent safety profiles observed.-Fineline Info & Tech