Rilzabrutinib

The global thrombocytopenia clinical trials landscape is expanding rapidly with over 25 pharmaceutical companies developing 25+ treatment therapies, according to recent market analysis.

• The FDA has approved Amgen's UPLIZNA (inebilizumab-cdon) as the first and only targeted treatment specifically for IgG4-related disease, following positive results from the Phase III MITIGATE trial. • The MITIGATE trial demonstrated an 87% reduction in disease flare risk compared to placebo, with the study enrolling 135 adults across 80 sites in 22 countries. • Multiple companies including Sanofi and Zenas BioPharma are advancing a robust pipeline of approximately 10 therapies for IgG4-related disease, potentially transforming treatment options for this rare inflammatory condition.

The FDA has granted orphan drug designation to Sanofi's rilzabrutinib for warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), two rare conditions with no currently approved treatments.

The FDA has granted orphan drug designation to Sanofi's rilzabrutinib for the treatment of autoimmune hemolytic anemia, highlighting potential new therapeutic options for this rare blood disorder.

• Sanofi will showcase 24 abstracts at AAAAI 2025, including new pooled results from Dupixent's phase 3 trials in chronic spontaneous urticaria and data across multiple inflammatory conditions. • New phase 2 data for rilzabrutinib, an investigational oral BTK inhibitor, will demonstrate its potential in treating moderate-to-severe chronic spontaneous urticaria patients. • The presentations will include four oral sessions and a late-breaking poster, covering diseases such as asthma, COPD, CRSwNP, and eosinophilic esophagitis.