Sanofi Secures EU Approval For Wayrilz, A First-Of-Its-Kind BTK Inhibitor For Immune Thrombocytopenia

The European Commission has approved Wayrilz (rilzabrutinib), a novel oral and reversible Bruton’s tyrosine kinase (BTK) inhibitor, as a treatment for adult patients with immune thrombocytopenia (ITP) who are refractory to existing therapies. This approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). Wayrilz offers a multi-immune modulation approach, targeting different pathways within the immune system to address the underlying causes of ITP, rather than simply focusing on increasing platelet counts. 

ITP is a complex autoimmune disorder characterized by low platelet levels, increased bleeding risk, and a range of often-overlooked physical and emotional symptoms that significantly impact patients’ quality of life. According to Dr. Waleed Ghanima, Head of Research and Consultant Hematologist at Østfold Hospital in Norway, traditional treatments mainly aim to restore platelet counts and reduce bleeding risk, but patients may still experience other burdensome symptoms. Wayrilz provides a new therapeutic approach that addresses multiple facets of the disease.

Sanofi emphasized that the European approval of Wayrilz reflects the company’s commitment to developing innovative therapies for rare and inflammatory diseases. Brian Foard, Executive Vice President and Head of Specialty Care at Sanofi, highlighted Wayrilz’s differentiated mechanism of action, which enables multi-immune modulation to target the root pathology of ITP and provide patients with an advanced treatment option to manage their condition more effectively. The approval was based on data from the pivotal LUNA 3 Phase 3 clinical trial (NCT04562766), which evaluated the efficacy and safety of Wayrilz compared to placebo in 202 adults with persistent or chronic ITP. 

The trial met all primary and secondary endpoints, demonstrating significant improvements in sustained platelet counts as well as other ITP-related symptoms. Key findings included a durable platelet response at week 25 in 23% of patients receiving Wayrilz compared to 0% in the placebo group, a faster time to first platelet response (36 days versus not reached in the placebo arm), and a longer duration of platelet response (7 weeks versus 0.7 weeks). Patients treated with Wayrilz also reported a 10.6-point improvement in overall quality of life, compared to a 2.3-point increase in the placebo group, based on the Immune Thrombocytopenia Patient Assessment Questionnaire.

The most common adverse reactions reported in the trial, occurring in at least 10% of patients, were diarrhea, nausea, headache, abdominal pain, and COVID-19. Wayrilz is already approved for ITP in the United States and the United Arab Emirates and is currently under regulatory review in Japan and China. The therapy has received multiple designations recognizing its potential to address rare diseases, including fast track and orphan drug designations in the U.S. for ITP, as well as orphan drug designations in the EU and Japan. 

Additionally, Wayrilz has received orphan drug and fast track designations for other rare conditions, including warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease. With this European approval, Wayrilz represents an important new treatment option for patients with ITP, providing a therapy that targets the underlying disease mechanisms and addresses the complex challenges associated with this rare autoimmune disorder.