Sanofi has received European Commission approval for Wayrilz (rilzabrutinib), a novel, oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, as a treatment for immune thrombocytopenia (ITP) in adults refractory to other therapies. This approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). Wayrilz addresses ITP through multi-immune modulation, targeting multiple immune pathways to tackle the underlying causes of the disease.
The European approval is based on the pivotal LUNA 3 phase 3 study (NCT04562766), which evaluated the efficacy and safety of Wayrilz compared to placebo in adults with persistent or chronic ITP. Patients who achieved platelet count response at 12 weeks continued through a 24-week double-blind period, during which 64% of Wayrilz patients and 32% of placebo patients maintained platelet count responses. The study also measured quality of life using the Immune Thrombocytopenia Patient Assessment Questionnaire, with Wayrilz recipients reporting a 10.6-point improvement compared to 2.3 points in the placebo group.
Wayrilz has already been approved in the United States and the UAE and is under regulatory review in Japan and China. In the US, it has received fast track and orphan drug designations for ITP, with similar orphan designations in the EU and Japan. Additional ongoing studies include rare diseases such as warm autoimmune haemolytic anaemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD), for which the drug has received FDA orphan designation and fast track status, as well as EU orphan designation for IgG4-RD.
The LUNA 3 study included oral Wayrilz 400 mg twice daily or placebo over 12 to 24 weeks, followed by a 28-week open-label period and a four-week safety follow-up or long-term extension. The primary endpoint for EU approval was the proportion of adults achieving platelet counts ≥50,000/µL for at least eight of the final 12 weeks of the 24-week blinded treatment period without rescue therapy. Secondary endpoints included time to platelet response, duration of response, need for rescue therapy, fatigue, and bleeding scores. The adolescent cohort is ongoing.
ITP is caused by complex immune dysregulation, leading to low platelet counts (<100,000/µL) and increased risk of bleeding and thromboembolic events. Patients may experience fatigue, cognitive impairment, and a reduced quality of life beyond physical symptoms.
Wayrilz’s approval in Europe underscores Sanofi’s commitment to developing innovative therapies for rare and inflammatory diseases, offering a new treatment option that targets the disease’s underlying pathology while improving patient outcomes.
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