Sanofi’s Wayrilz gains approval in Japan for the treatment of immune thrombocytopenia

Japan’s Ministry of Health, Labour and Welfare has granted marketing and manufacturing authorisation for Wayrilz, a novel oral reversible Bruton’s tyrosine kinase inhibitor, for the management of persistent or chronic immune thrombocytopenia in patients whose response to other treatments has been insufficient or where tolerability has presented difficulties. By acting across multiple pathways within the immune system through multi-immune modulation, Wayrilz targets the root mechanisms driving the disease.

Evidence from the LUNA 3 Phase 3 study forms the basis of the approval, with Wayrilz clearing both primary and secondary endpoints and demonstrating a positive effect on sustained platelet counts as well as broader disease symptoms.

“For ITP patients, achieving platelet counts close to normal and living without worrying about bleeding is extremely important. Additionally, in recent years, there has been growing interest in various other symptoms associated with ITP such as fatigue,” said Hisashi Kato, MD, PhD, Department of Blood Transfusion and Cell Therapy, The University of Osaka Hospital, Suita, Japan. “Wayrilz is a novel BTKi that works through multi-immune modulation to help address the root cause of ITP pathophysiology. It is expected to become a new therapeutic option that may help patients with disease management, including not only improvement in platelet counts but also considering patients’ quality of life.”

“People living with immune thrombocytopenia who continue to experience symptoms, despite trying existing treatments, face a great unmet need,” said Manuela Buxo, Executive Vice President, Global Head of Specialty Care at Sanofi. “Wayrilz has a differentiated mechanism of action that enables multi-immune modulation to address the underlying pathophysiology of ITP and offers new hope to patients who have not responded to prior therapy. This milestone underscores Sanofi’s commitment to bringing innovative treatment options to rare disease patients around the world.”

The LUNA 3 Phase 3 study examined the efficacy and safety of Wayrilz against placebo in 202 adults with persistent or chronic ITP. Those who achieved a platelet count response at week 12 were eligible to continue through the full 24-week double-blind period, a threshold met by 64% of those in the Wayrilz arm and 32% of those receiving placebo. Against the placebo group, patients treated with Wayrilz achieved the following:

• A statistically significant durable platelet response at week 25, recorded in 23% of those on Wayrilz compared to none in the placebo group
• A faster time to first platelet response, reached at a median of 36 days in the Wayrilz arm versus a threshold that was never reached in the placebo group
• A longer overall duration of platelet response, with a least squares mean of 7 weeks on Wayrilz against 0.7 weeks with placebo

Patients on Wayrilz also reported a 10.6-point improvement in overall quality of life as measured by the Immune Thrombocytopenia Patient Assessment Questionnaire, a validated tool for capturing ITP-related symptoms and their impact, compared to a 2.3-point gain among those on placebo. These findings are descriptive in nature and the analysis was not powered to reach statistical significance.

Among the most frequently occurring adverse reactions, each arising in at least 10% of patients, were diarrhoea, nausea, headache, abdominal pain, and COVID-19.

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