Rilzabrutinib

Europe's Committee for Medicinal Products for Human Use has turned down Sanofi's Rezurock while also offering a positive opinion for the company's thrombocytopenia treatment Wayrilz. Europe’s Committee for Medicinal Products for Human Use (CHMP) has given a thumbs-down (PDF) to Sanofi’s Rezurock as a third-line treatment for chronic graft-versus-host disease (cGVHD).The European Medicines Agency’s panel of experts issued its negative opinion because it “was difficult to quantify” the effect Rezurock had on patients relative to other treatments. Additionally, the CHMP noted that a study in which patients used Rezurock as a first-line treatment “did not show it had any beneficial effects.”The CHMP also stated that due to the need for new treatments in this indication, it considered recommending Rezurock for a conditional marking nod. But the regulator ultimately decided not to, given that Sanofi isn't in a position to provide more efficacy data on the drug until 2030.The company plans to seek a reexamination of the opinion.“Sanofi is confident in the body of clinical and real-world evidence that underscores Rezurock’s consistent efficacy and well-established safety profile,” Olivier Charmeil, Sanofi’s executive vice president of general medicines, said in a release. “We will continue to work closely with the European Medicines Agency with the aim of bringing this treatment to patients in the EU who are waiting.” Out of 93 recommendations this year on new medicines, the Rezurock snub represents the sixth negative opinion from the CHMP so far. Sanofi gained Rezurock in a $1.9 billion buyout of Kadmon in 2021, less than two months after the FDA approved the treatment for third-line use in cGVHD. Today, the medicine is available in 20 countries, including the U.K., Canada and China.More than 17,000 patients have been prescribed Rezurock since its initial U.S. nod, Sanofi said Friday. Sales of the treatment reached 132 million euros ($154 million) in the first half of this year, which was a 21% increase over the same period in 2024.A whirlwind of endorsements It wasn't all bad news for Sanofi this week, however, as the CHMP recommended the company’s Bruton tyrosine kinase (BTK) inhibitor Wayrilz for approval to treat immune thrombocytopenia in adult patients who are refractory to other therapies.The positive opinion comes after the FDA approved the potential blockbuster in August. Sanofi gained the drug—along with another BTK hopeful tolebrutinib—in 2020 through its $3.7 billion acquisition of Principia Biopharma.The CHMP also signed off on Insmed’s first-in-class treatment Brinsupri for non-cystic fibrosis bronchiectasis. The recommendation comes two months after the FDA endorsed the dipeptidyl peptidase 1 (DPP1) inhibitor. The oral treatment is indicated for patients ages 12 and older who have had at least two flare-ups of the lung disorder over the previous year. Apart from the new drug endorsements, the CHMP issued further positive opinions this week, which would expand the label for eight previously approved drugs, including Pfizer’s COVID antiviral Paxlovid, Johnson & Johnson’s autoimmune treatment Tremfya, Regeneron’s skin and lung cancer therapy Libtayo, Agios’ anemia drug Pyrukynd and CStone’s lung cancer medicine Cejemly. Also making the grade were the following blood cancer drugs: Bristol Myers Squibb’s Breyanzi, Roche’s Gazyvaro and Novartis’ Scemblix.

Sanofi’s Wayrilz recommended for EU approval by the CHMP to treat immune thrombocytopenia Recommendation based on LUNA 3 phase 3 study demonstrating rapid and durable platelet response and significant improvements in bleeding, quality of life measures, and other ITP symptoms If approved, Wayrilz will be the first BTK inhibitor for ITP in the EU, targeting the root cause of the disease through multi-immune modulation Paris, October 17, 2025. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Wayrilz (rilzabrutinib) as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. A final decision is expected in the coming months. “The CHMP’s recommendation of Wayrilz for approval is a positive step forward for people in the EU living with ITP who continue to experience symptoms that impact their daily living despite treatment with certain other therapies,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “Through multi-immune modulation, Wayrilz shows promise in addressing the root cause of ITP – complex immune system dysregulation, reinforcing Sanofi’s commitment to finding novel ways to address unmet patient needs in rare and immunological diseases.” The positive CHMP opinion is based on the pivotal LUNA 3 phase 3 study (clinical study identifier: NCT04562766), presented at the 66th American Society of Hematology Annual Meeting and Exposition in December 2024 and also published in Blood. Wayrilz met both the primary and secondary endpoints, making LUNA 3 the first phase 3 study to show a positive impact on sustained platelet counts, bleeding, and other ITP symptoms with an oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor. Wayrilz has already been approved in the US and the United Arab Emirates. In addition to the EU, Wayrilz is currently under regulatory review for ITP in China. It received fast track and orphan drug designations (ODD) in the US for ITP, with similar orphan designations in the EU and Japan. In other indications under investigation, the US Food and Drug Administration (FDA) granted Wayrilz ODD for three additional rare diseases, including warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz also received FDA fast track designation and EU orphan designation in IgG4-RD. About the LUNA 3 studyLUNA 3 (clinical study identifier: NCT04562766) was a randomized, multicenter, phase 3 study evaluating the efficacy and safety of Wayrilz vs. placebo in adult and adolescent patients with persistent or chronic ITP. Patients received either oral Wayrilz 400 mg twice a day or placebo through a 12- to 24-week double-blind treatment period, followed by a 28-week open-label treatment period, and then a four-week safety follow-up or long-term extension period. The adolescent part of the study is ongoing. The primary endpoint for the EU is the proportion of adult participants able to achieve platelet counts at or above 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. Secondary endpoints included time to platelet response (platelet count ≥50 x 109/L or between 30 x 109/L and <50 x 109/L and at least doubled from baseline in absence of rescue therapy), number of weeks maintaining a specific platelet response (i.e., doubled or within range), rescue therapy use, physical fatigue score, and bleeding score as assessed by change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25. About WayrilzWayrilz (rilzabrutinib) is the first BTK inhibitor for ITP that helps address the root cause of disease through multi-immune modulation. This innovative therapy is approved in the US for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Additionally, Wayrilz is approved in the United Arab Emirates for adult patients with persistent or chronic ITP who have had an insufficient response or intolerance to a previous treatment. 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, Wayrilz can selectively inhibit the BTK target. Wayrilz is being studied across a variety of rare diseases, including wAIHA, IgG4-RD, and SCD. These additional indications are currently under investigation and have not been approved by regulatory authorities. About ITPITP is a disease of complex immune dysregulation that causes low platelet counts (<100,000/μL), resulting in a variety of bleeding symptoms and thromboembolism risk. Beyond bruising and bleeding, which can include potentially life-threatening episodes like intracranial hemorrhage, people living with ITP may experience reduced quality of life, including physical fatigue and cognitive impairment. 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 Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com Evan Berland | +1 215 432 0234 | evan.berland@sanofi.comLéo Le Bourhis | + 33 6 75 06 43 81 | leo.lebourhis@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.comTimothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.comLéa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com Investor RelationsThomas Kudsk Larsen | +44 7545 513 693 | thomas.larsen@sanofi.comAlizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com Felix 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.com Tarik Elgoutni | +1 617 710 3587 | tarik.elgoutni@sanofi.comThibaud Châtelet | +33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Yun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com Sanofi forward-looking statementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. 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