The Ministry of Health, Labour and Welfare in Japan has granted marketing and manufacturing authorization to Sanofi for Wayrilz (rilzabrutinib) to treat persistent or chronic immune thrombocytopenia (ITP) in adults. This approval addresses a significant unmet need for patients who do not respond sufficiently to other treatments or face tolerability issues. Wayrilz is a novel oral reversible Bruton’s tyrosine kinase inhibitor (BTKi) that works through multi-immune modulation to target key pathways across the immune system.

The regulatory decision is based on the LUNA 3 phase 3 study (clinical study identifier: NCT04562766). In this trial, Wayrilz met the primary and secondary endpoints, demonstrating a positive impact on sustained platelet counts and other symptoms compared to placebo. The study evaluated the efficacy and safety of the drug in adults with persistent or chronic ITP.

LUNA 3 Phase 3 Study Results

The study included 202 adults, with patients eligible to continue the full 24-week double-blind period if they achieved a platelet count response at 12 weeks. Results showed that 64% of patients in the Wayrilz arm continued, compared to 32% in the placebo arm. Key outcomes for patients receiving Wayrilz included:

Patients receiving Wayrilz reported an overall 10.6-point improvement in the quality of life domain, compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire. The most common adverse reactions, with an incidence of 10% or more, were diarrhea, nausea, headache, abdominal pain, and COVID-19.

Future Indications and Designations

Wayrilz is currently being studied across multiple other rare diseases, including IgG4-related disease (IgG4-RD), warm autoimmune hemolytic anemia (wAIHA), and sickle cell disease. These additional indications are under investigation and have not yet received regulatory approval. Japan has granted Wayrilz orphan drug designation for ITP, IgG4-RD, and wAIHA.

Sanofi today announced the appointment of Paulo Fontoura, MD, PhD, FAAN, as Executive Vice President, Global Head of Research & Development Pharma, effective September 1, 2026. Based in Paris, Fontoura will join Sanofi's Executive Committee and report to Chief Executive Officer Belén Garijo. He succeeds Dr. Houman Ashrafian, who is leaving the company to pursue an external opportunity.

In his new role, Fontoura will lead Sanofi's end-to-end innovation engine, encompassing research, translational medicine, clinical development, and regulatory affairs. His primary responsibility will be advancing a differentiated pipeline and accelerating the delivery of transformative medicines to patients. The appointment comes as Sanofi continues its R&D transformation and progresses a pipeline of new medicines across multiple therapeutic areas.

Fontoura brings more than 25 years of experience in academic medicine, translational science, clinical development, and pharmaceutical innovation. Most recently, he served as Chief Medical Officer of Xaira Therapeutics, an AI-native biotechnology company. Prior to that, he spent over 15 years at Roche, where he held senior leadership positions, including Senior Vice President and Global Head of Clinical Development for Neuroscience, Immunology, Ophthalmology, Infectious and Rare Diseases.

Paulo Fontoura's Profile

During his tenure at Roche, Fontoura oversaw a global organization of physicians and clinical scientists and managed a broad late-stage portfolio. His teams contributed to the development of over 60 new molecular entities, leading to over 20 FDA and EMA first approvals and line extensions. He is a board-certified neurologist and a Fellow of the American Academy of Neurology, having authored more than 80 peer-reviewed scientific publications.

Belén Garijo, Chief Executive Officer of Sanofi, expressed confidence in the new appointment, highlighting Fontoura's respected leadership and strong track record across research, development, and innovation. Fontoura stated he is thrilled to join Sanofi at an exciting moment, citing the company's leadership position in immunology and its commitment to scientific innovation and AI-powered transformation.