Press Release: Sanofi’s Wayrilz recommended for EU approval by the CHMP to treat immune thrombocytopenia

Sanofi's Wayrilz (rilzabrutinib) has received a positive opinion from the European Medicines Agency's CHMP for approval to treat immune thrombocytopenia (ITP) in adult patients refractory to other treatments. This recommendation, based on the pivotal LUNA 3 phase 3 study demonstrating rapid and durable platelet response and improved patient outcomes, positions Wayrilz as the first BTK inhibitor for ITP in the EU, targeting the disease's root cause. The drug is already approved in the US and UAE, and is under review in China, indicating significant global market expansion potential for Sanofi in rare and immunological diseases.

Analysis

Sanofi's Wayrilz (rilzabrutinib) has received a strongly positive opinion from the European Medicines Agency's CHMP for approval in the EU to treat immune thrombocytopenia (ITP) in adult patients refractory to other treatments. This recommendation is based on the pivotal LUNA 3 phase 3 study, which demonstrated rapid and durable platelet response and significant improvements in bleeding and quality of life measures. If approved, Wayrilz will be the first BTK inhibitor for ITP in the EU, addressing the disease's root cause through multi-immune modulation. This regulatory milestone, coupled with existing approvals in the US and UAE and ongoing regulatory review in China, signals significant global market expansion potential for Sanofi in rare and immunological diseases.

Wayrilz has already secured fast track and orphan drug designations in the US, EU, and Japan for ITP, highlighting its perceived medical necessity and potential for expedited market access. Furthermore, the drug is under investigation for additional rare diseases like warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD), with some already receiving FDA orphan drug and fast track designations, indicating a broader pipeline value.

The successful LUNA 3 study, which met both primary and secondary endpoints, provides robust clinical validation for Wayrilz's efficacy and safety profile. A final EU decision is expected in the coming months, which could open a new substantial revenue stream for Sanofi and reinforce its position in the specialty care market. This development aligns with Sanofi's commitment to finding novel ways to address unmet patient needs in rare and immunological diseases, enhancing its R&D-driven biopharma strategy.

Key Decisions for Investors

• Monitor the final EU approval decision for Wayrilz in the coming months, as this represents a critical catalyst for Sanofi's specialty care segment and potential revenue growth.
• Evaluate the potential market penetration and revenue projections for Wayrilz, considering its first-in-class BTK inhibitor status for ITP in the EU and existing global approvals in the US and UAE.
• Assess Sanofi's pipeline strength and long-term growth trajectory, particularly regarding Wayrilz's additional rare disease indications currently under investigation, which could provide future upside and diversify revenue streams.