Sanofi announced that the European Commission has granted marketing authorization for Wayrilz (rilzabrutinib) to treat adult patients with immune thrombocytopenia (ITP) who have not responded to other therapies. The approval, effective December 23, 2025, follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
Wayrilz is a first‑in‑class, oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor that delivers multi‑immune modulation by targeting key pathways involved in platelet destruction and immune dysregulation. The drug’s mechanism allows it to address the underlying immune pathology of ITP, offering a novel therapeutic option that is not available in the EU market.
The pivotal LUNA 3 phase‑3 study, which enrolled 202 patients, demonstrated that 23 % of Wayrilz recipients achieved a durable platelet response at week 25 compared with 0 % of placebo patients. The median time to response was 36 days for Wayrilz versus 0 days for placebo, and the median duration of response was 7 weeks versus 0.7 weeks. These results met both primary and secondary endpoints and were presented at the 66th American Society of Hematology Annual Meeting and published in the journal Blood, underscoring the drug’s efficacy and safety profile.
From a business perspective, the EU approval expands Sanofi’s specialty‑care portfolio into a new rare‑disease indication and positions the company to capture a share of the growing ITP market, which is expected to expand as awareness increases and treatment options diversify. Wayrilz is already approved in the United States and the United Arab Emirates, and regulatory reviews are underway in Japan and China, suggesting a broader global launch trajectory. The approval also reinforces Sanofi’s strategic focus on immunology and rare diseases, adding a first‑in‑class BTK inhibitor to its pipeline and opening potential for future label expansions into related indications such as warm autoimmune hemolytic anemia, IgG4‑related disease, and sickle cell disease.
Brian Foard, Executive Vice President and Head of Specialty Care at Sanofi, said the approval “underscores Sanofi’s commitment to leveraging our knowledge of the immune system to develop innovative treatments that make a meaningful impact on people living with rare and inflammatory diseases.” He added that Wayrilz’s differentiated mechanism “enables multi‑immune modulation to address the underlying pathology of ITP, allowing patients to benefit from an advanced treatment to help manage their disease.”
The approval also signals Sanofi’s continued investment in targeted therapies for complex autoimmune conditions, positioning the company to compete with emerging BTK inhibitors and other novel agents in the immunology space. With Wayrilz’s first‑in‑class status in the EU, Sanofi gains a competitive advantage that could translate into a new revenue stream and strengthen its specialty‑care positioning for the long term.
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