Improving DxMultiomics, Powered by Your Choices

We're building DxMultiomics to be The Central Nexus of Biotech Intelligence. To ensure a seamless experience and continuous improvement, we utilize website analytics. These tools help us monitor site performance, understand how users navigate the platform, and identify opportunities to make your research journey more efficient. We handle this data with the utmost respect for your privacy, and you can choose what works best for you.

Sanofi’s rilzabrutinib earns orphan designation in the EU for IgG4-related disease
_↗ sanofi.com
News Image

Sanofi announced that the European Medicines Agency (EMA) granted orphan designation to rilzabrutinib, an investigational drug for the treatment of IgG4-related disease (IgG4-RD). This designation is given to therapies that address rare, life-threatening, or debilitating conditions affecting fewer than 5 in 10,000 people in the EU. This new status highlights Sanofi's commitment to developing new medicines for immune-mediated rare diseases.

The orphan designation was supported by positive data from a Phase 2 study of rilzabrutinib in patients with IgG4-RD. The results, presented at the EULAR 2025 Congress, showed that after 52 weeks of treatment, the drug led to a significant reduction in disease flare-ups and other disease markers. The study also demonstrated that rilzabrutinib helped reduce the need for glucocorticoids, a common but often problematic treatment for the condition. The drug's safety profile in the study was consistent with previous trials, with no new safety signals observed.

IgG4-RD is a chronic, progressive, and relapsing immune-mediated rare disease that can affect almost any organ in the body. It can lead to irreversible organ damage and, in some cases, can be fatal.

The disease is characterized by frequent flare-ups, and patients often experience periods of exacerbated symptoms. In the U.S., it affects approximately 8 out of every 100,000 adults each year, though its global prevalence is unknown due to diagnostic challenges. The rarity and complexity of IgG4-RD make the development of new treatments a critical need.

Rilzabrutinib is a novel, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor. By working to restore immune balance, it has the potential to treat several rare immune-mediated diseases. This orphan designation for IgG4-RD in the EU is the latest in a series of similar designations for the drug. Rilzabrutinib has also received orphan designations for conditions such as immune thrombocytopenia (ITP) in the U.S., EU, and Japan, as well as for warm autoimmune hemolytic anemia and sickle cell disease in the U.S. It has also been granted fast track designation in the U.S. for both ITP and IgG4-RD.

Currently, rilzabrutinib is under regulatory review for ITP in the U.S., EU, and China. The FDA's decision for ITP is expected by August 29, 2025.

Log In
Sign Up