Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease
Rilzabrutinib granted orphan drug designation in the US for sickle cell disease
Fourth orphan drug designation for rilzabrutinib in rare diseases
Under regulatory review in the US, the EU, and China in immune thrombocytopenia
Paris, June 3, 2025. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation, in sickle cell disease. The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the US.
Global Head of Development, Rare Diseases'Receiving our fourth orphan drug designation for rilzabrutinib reinforces our continued dedication to developing medicines to address the unmet medical needs of people living with rare diseases. People with sickle cell disease often live with severe episodes of pain from vaso-occlusive crises and other complications that can significantly impact both quality of life and life expectancy. There remains a need for novel treatment approaches to address these experiences by modulating the immune system responses that can contribute to sickle cell disease pathogenesis.'
In addition to sickle cell disease, rilzabrutinib has received orphan drug designation for immune thrombocytopenia (ITP) in the US, the EU, and Japan, for warm autoimmune hemolytic anemia (wAIHA) in the US and the EU, and for IgG4-related disease (IgG4-RD) in the US.
The safety and efficacy of rilzabrutinib have not been determined by any regulatory authority. Rilzabrutinib is currently under regulatory review in the US, the EU, and in China for its potential use in ITP. The target action date for the FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025.
Sickle cell disease supporting dataPreclinical data on sickle cell disease was presented at ASH 2024 showing that rilzabrutinib helped reduce vaso-occlusion –blockage of blood vessels – and inflammation in transgenic mice with sickle cell disease.
About rilzabrutinib Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (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 Sanofi's TAILORED COVALENCY® technology, rilzabrutinib can selectively inhibit the BTK target while potentially reducing the risk of off-target side effects.
About sickle cell diseaseSickle cell disease is a group of rare, genetic blood disorders in which red blood cells are misshapen, typically in a sickle or crescent shape, causing them to get stuck in small blood vessels, blocking blood flow. This can lead to episodes of severe pain as well as other health complications including infections, stroke, lung, eye, and kidney disease. Sickle cell disease affects more than 100,000 people in the US, approximately 90% of whom are African American and 3-9% of whom are Hispanic or Latino. Approximately 1 in 365 babies of African American descent born in the US have sickle cell disease, while an estimated 1 in 13 are carriers of the disease. People in the US with sickle cell disease have an estimated life expectancy that is 20 years shorter than average.
About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and creating 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
Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.com Léo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.com Victor Rouault | +33 6 70 93 71 40 | victor.rouault@sanofi.com Timothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.com
Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | thomas.larsen@sanofi.com Alizé Kaisserian | +33 6 47 04 12 11 | alize.kaisserian@sanofi.comFelix Lauscher | +1 908 612 7239 | felix.lauscher@sanofi.comKeita Browne | +1 781 249 1766 | keita.browne@sanofi.com Nathalie Pham | +33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik Elgoutni | +1 617 710 3587 | tarik.elgoutni@sanofi.com Thibaud Châtelet | +33 6 80 80 89 90 | thibaud.chatelet@sanofi.comYun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com
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.
All trademarks mentioned in this press release are the property of the Sanofi group.
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