Sanofi’s Wayrilz FDA Approval Brings New Oral BTK Inhibitor for ITP

The U.S. Food and Drug Administration (FDA) has granted approval to Sanofi’s (NASDAQ: SNY) Wayrilz (rilzabrutinib) for the treatment of adult patients with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to prior therapy. The decision is rooted in the pivotal Phase 3 LUNA 3 study, which demonstrated that Wayrilz achieved both its primary and secondary endpoints, producing durable platelet count gains and meaningful relief of ITP symptoms.

Key Highlights

• First‑in‑class, oral, reversible BTK inhibitor
• Multi‑immune modulation – targets several pathways that drive platelet destruction
• Rapid, sustained platelet response with a favorable safety profile

Mechanism of Action

Wayrilz selectively blocks Bruton’s tyrosine kinase (BTK), a pivotal enzyme in B‑cell receptor signaling. By interrupting BTK activity, the drug dampens autoreactive B‑cell and macrophage activity, thereby reducing platelet clearance. Its reversible binding allows for precise control of immune modulation, minimizing off‑target effects.

Regulatory Milestones

Future Directions

Wayrilz is currently under investigation in several other rare immune‑mediated disorders where BTK inhibition may be therapeutic, including:

• Warm autoimmune hemolytic anemia
• IgG4‑related disease
• Sickle cell disease

These trials aim to extend the drug’s impact beyond ITP, leveraging its broad immune‑modulatory profile.-Fineline Info & Tech