Sanofi’s Rilzabrutinib Receives Breakthrough and Orphan Designations for Warm Autoimmune Hemolytic Anemia

(IN BRIEF) Sanofi has received Breakthrough Therapy designation from the U.S. FDA and orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for rilzabrutinib (Wayrilz) in warm autoimmune hemolytic anemia, based on data from the ongoing LUMINA 2 and LUMINA 3 clinical trials. The recognitions reflect the significant unmet need in wAIHA, a rare immune disorder with no approved disease-targeting treatments, while reinforcing rilzabrutinib’s broader potential across multiple rare diseases such as ITP, IgG4-related disease, and sickle cell disease, where it already holds various regulatory designations.

(PRESS RELEASE) PARIS, 9-Feb-2026 — /EuropaWire/ — Sanofi has secured important regulatory recognitions for rilzabrutinib (Wayrilz) in the treatment of warm autoimmune hemolytic anemia (wAIHA), underscoring the therapy’s potential to address a serious rare immune disorder with limited treatment options. The U.S. Food and Drug Administration has granted the medicine Breakthrough Therapy designation, while Japan’s Ministry of Health, Labour and Welfare has awarded it orphan drug status for the same indication.

These regulatory milestones are supported by clinical evidence generated from Sanofi’s ongoing development programme, including data from the LUMINA 2 phase 2b study and the continuing LUMINA 3 phase 3 trial. LUMINA 2 has assessed the safety and efficacy of rilzabrutinib in patients with wAIHA, while LUMINA 3 is currently comparing the investigational therapy against placebo in a larger patient population. Both studies are designed to evaluate whether rilzabrutinib can meaningfully improve outcomes in a condition for which there are currently no approved treatments targeting the underlying immune dysfunction.

Warm autoimmune hemolytic anemia is a rare but potentially life-threatening disease in which the immune system mistakenly attacks and destroys red blood cells, often leading to chronic anemia, severe fatigue, and risk of serious complications. The absence of disease-specific therapies has created a significant unmet medical need, which Sanofi aims to address through its BTK inhibitor programme.

The FDA’s Breakthrough Therapy designation is intended to accelerate the development and review of medicines that show promising early evidence of substantial clinical improvement over existing options for serious or life-threatening conditions. Meanwhile, Japan’s orphan drug designation recognises rilzabrutinib’s potential to benefit a small patient population with high unmet need, while providing regulatory incentives to support further development.

Karin Knobe, Global Head of Development for Rare Diseases at Sanofi, said that these recognitions highlight the ongoing unmet need faced by people living with wAIHA. She added that the designations reinforce Sanofi’s commitment to advancing innovative therapies for rare diseases where treatment options remain limited or non-existent.

Rilzabrutinib is already approved in the United States, the European Union, and the United Arab Emirates under the brand name Wayrilz for the treatment of adults with immune thrombocytopenia (ITP), another rare immune-mediated disorder. Regulatory review for its use in ITP is currently underway in Japan. Sanofi emphasised that, outside of these approved indications, all other potential uses of rilzabrutinib remain investigational and have not yet been evaluated by regulatory authorities.

Beyond wAIHA and ITP, rilzabrutinib has received multiple regulatory designations that reflect its broader therapeutic potential. The FDA has previously granted orphan drug status for autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease, as well as fast track designation for ITP and IgG4-RD. In Europe, the medicine has also received orphan designation for ITP, autoimmune hemolytic anemia, and IgG4-RD.

Warm autoimmune hemolytic anemia accounts for more than half of all autoimmune hemolytic anemia cases and is characterised by the premature destruction of red blood cells due to autoantibodies. In the United States and European Union, the condition is estimated to affect between four and 24 people per 100,000, while in Japan the prevalence is even lower at approximately three to 10 people per million. Symptoms can include debilitating fatigue, dizziness, palpitations, and shortness of breath, with some patients also facing an elevated risk of thromboembolic events.

Rilzabrutinib is a novel, oral, reversible covalent inhibitor of Bruton’s tyrosine kinase, a key enzyme involved in multiple immune and inflammatory pathways. By selectively targeting BTK through Sanofi’s TAILORED COVALENCY® technology, the therapy is designed to restore immune balance across several rare immune-mediated and inflammatory diseases. BTK is expressed in B cells, macrophages, and other innate immune cells, making it a central driver of disease activity in conditions such as wAIHA, ITP, IgG4-RD, and sickle cell disease.

Sanofi continues to evaluate rilzabrutinib across a range of rare diseases in clinical development programmes, with the aim of bringing new targeted treatment options to patients with serious immune disorders.

About wAIHA
wAIHA is a rare, potentially life-threatening, autoimmune disorder rooted in complex immune system dysregulation. It represents more than half of autoimmune hemolytic anemia cases. In wAIHA, autoantibodies lead to the premature destruction of the body’s own red blood cells (hemolysis), sometimes faster than the bone marrow can replace them. In the US and EU, autoimmune hemolytic anemia is estimated to affect four to 24 people out of 100,000, while in Japan it is rarer, affecting three to 10 people per million. People living with wAIHA may experience debilitating fatigue, dizziness, palpitations, and shortness of breath, and may face complications such as thromboembolism.

About rilzabrutinib
Rilzabrutinib, Wayrilz where approved, is a novel, oral, reversible covalent BTK inhibitor that has the potential to be an effective new medicine for several rare immune-mediated or inflammatory diseases by working to restore immune balance via multi-immune modulation. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways. With the application of the TAILORED COVALENCY® technology, rilzabrutinib can selectively inhibit the BTK target. Wayrilz is now approved for the treatment of immune thrombocytopenia (ITP) in the US, the EU, and the UAE. Regulatory review for use in ITP is currently ongoing in Japan.

In addition to ITP and wAIHA, rilzabrutinib is being studied across a variety of rare diseases, including IgG4-RD and SCD. These additional indications are currently under investigation and have not been approved by regulatoryauthorities.

About Sanofi
Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time.

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Sanofi forward-looking statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Sanofi’s Rilzabrutinib Receives Breakthrough and Orphan Designations for Warm Autoimmune Hemolytic Anemia

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