Fitusiran

Sanofi’s Qfitlia and Cablivi approved in China, expanding care for rare diseases Qfitlia, the first antithrombin-lowering therapy for hemophilia, can offer consistent protection with as few as six injections a yearCablivi, the first Nanobody medicine, targets acquired/immune-mediated thrombotic thrombocytopenic purpura — a rare, life-threatening blood clotting disorder Paris, December 11, 2025. The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rare hematologic diseases: Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura. These approvals mark another step in Sanofi’s long-term commitment to China, reinforcing the company’s ambition to bring transformative medicines across diverse disease areas. With Qfitlia and Cablivi, Sanofi reaches its fourth and fifth approvals in China this year, following Tzield for stage 2 type 1 diabetes and Sarclisa for two indications in relapsed and newly diagnosed multiple myeloma. Qfitlia is the first antithrombin (AT)-lowering therapy for routine prophylaxis in people with hemophilia. Qfitlia is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in pediatric patients 12 years of age and older, and adults with severe hemophilia A (coagulation factor VIII deficiency, FVIII<1%) with or without factor VIII inhibitors, or severe hemophilia B (coagulation factor IX deficiency, FIX<1%) with or without factor IX inhibitors. This approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous injections, and low-volume dosing. Hemophilia affects more than 40,000 people in China. Cablivi is the first Nanobody targeted therapy designed to treat acquired/immune-mediated thrombotic thrombocytopenic purpura (aTTP/iTTP) in adults and adolescents aged 12 or older weighing at least 40 kg. This approval brings an innovative medicine specifically indicated for this rare and life-threatening blood clotting disorder to China, where approximately 2700 patients are diagnosed annually. Despite standard treatments, aTTP/iTTP carries a mortality rate of up to 20%. Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis, and is designed to inhibit the interaction between vWF and platelets. Used in conjunction with plasma exchange and immunosuppressive therapy, it helps by inhibiting the formation of microthrombi, which contribute to organ damage during the course of the disease. These two approvals expand Sanofi's rare hematology portfolio in China, addressing critical unmet needs across both chronic bleeding disorders and acute clotting emergencies. “Qfitlia represents a potentially transformative advancement for the hemophilia community in China, shifting care from treating bleeds as they occur to helping prevent them altogether. By offering effective bleed protection and simplified administration, Qfitlia has the potential to make prophylaxis more accessible for people with hemophilia worldwide,” said Brian Foard, Executive Vice President, Head of Specialty Care, Sanofi. “Cablivi addresses a critical unmet need for patients facing aTTP/iTTP. Together, these approvals highlight Sanofi's commitment to delivering meaningful innovation and improving outcomes for people living with rare diseases in China and around the world.” In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include: Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand (estimated mean: ABR 9.0 vs. 31.4, respectively; p<.0001) and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors (estimated mean: ABR 5.1 vs. 19.1, respectively; p<0.0006)Median observed ABR during the open-label extension study was 3.8 (interquartile range (IQR): 0.0–11.2) in patients without inhibitors and 1.9 (IQR: 0.0–5.6) in patients with inhibitorsNearly half of patients in the open-label extension study experienced one or fewer bleeds (47% 0-1 bleeds and 31% 0 bleeds)Nearly 80% of participants were on a regimen of six injections per year by the conclusion of the open-label extension study, and 94% achieved target AT levels with 0-1 dose adjustments “The approval of Qfitlia marks a true transition into a new era of non-factor prophylactic treatment for hemophilia in China. Requiring potentially just six subcutaneous injections annually, it significantly reduces disease burden, eliminating the need for frequent intravenous injections associated with traditional factor therapy,” said Sun Jing, Chief Physician of Hematology, Nanfang Hospital at Southern Medical University, Guangzhou, China. “By lowering antithrombin to restore coagulation balance, this innovation offers people living with hemophilia A or B, with or without inhibitors, a novel treatment option.” Serious thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity have occurred in Qfitlia-treated patients. The most common adverse reactions (incidence >10%) are viral infection, nasopharyngitis, and bacterial infection. About hemophiliaHemophilia A and B are rare, congenital, lifelong, bleeding disorders in which the ability of a person’s blood to clot is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and significantly impact quality of life. Hemophilia A and B are caused by a deficiency of factor VIII and IX, respectively, resulting in insufficient thrombin generation and ineffective clot formation, which is further complicated in people who develop inhibitors to their factor treatment. About aTTPAcquired thrombotic thrombocytopenic purpura (aTTP, also known as immune-mediated thrombotic thrombocytopenic purpura (iTTP) is an ultra-rare life-threatening autoimmune-based blood clotting disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to thrombocytopenia (low platelet count); microangiopathic hemolytic anemia (loss of red blood cells through destruction); ischemia (restricted blood supply to parts of the body); and widespread organ damage, especially in the brain and heart. About the ATLAS clinical development programThe efficacy and safety of Qfitlia is being investigated in the ATLAS clinical development program. The program includes completed phase 3 studies ATLAS-INH (clinical study identifier: NCT03417102), ATLAS-A/B (clinical study identifier: NCT03417245), and ATLAS-PPX (clinical study identifier: NCT03549871). There are three ongoing phase 3 studies ATLAS-NEO (clinical study identifier: NCT05662319), ATLAS-PEDS (clinical study identifier: NCT03974113), and ATLAS OLE (clinical study identifier: NCT03754790). The ongoing ATLAS-OLE study is a single-arm, phase 3, open-label study evaluating the safety and efficacy of Qfitlia with a revised AT dosing regimen (AT-DR), which was designed to maintain an AT target range of 15%-35% in patients who have completed a prior phase 3 ATLAS clinical trial. This study includes lower doses and less-frequent dosing than earlier studies of Qfitlia. The efficacy of Qfitlia AT-DR treatment was assessed by comparing the AT-DR treatment data from ATLAS-OLE to the control data from studies ATLAS-INH and ATLAS-A/B. The analyses follow the intent to treat principle. About QfitliaQfitlia (fitusiran) is a first-in-class AT-lowering therapy approved by the NMPA. Qfitlia prevents bleeds and helps rebalance hemostasis by lowering AT, a protein that inhibits blood clotting, to promote thrombin generation. Qfitlia is a small interference RNA therapeutic that utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc conjugate technology. The US Food and Drug Administration (FDA) approved Qfitlia on March 28, 2025, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. Additional submissions for Qfitlia are under review with regulatory authorities around the world. About Cablivi Cablivi (caplacizumab) is a bivalent anti-von Willebrand Factor (vWF) Nanobody VHH, used in conjunction with plasma exchange and immunosuppressive therapy, for the treatment of patients experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), also known as immune-mediated thrombotic thrombocytopenic purpura (iTTP). Cablivi is the first and only treatment targeted to block the formation of microthrombi, small blood clots that form in the microvasculature, helping prevent organ damage. Cablivi is currently available in nearly 30 countries including the US, the EU, UK, Switzerland, Brazil, Colombia, Japan, and five Greater Gulf region states. Cablivi earned priority review for its approval in China, as well as priority review designation from the FDA for a pending label expansion to include the treatment of adolescents aged 12 years and older. 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.comEvan 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.comFelix Lauscher | +1 908 612 7239 | felix.lauscher@sanofi.comKeita Browne | +1 781 249 1766 | keita.browne@sanofi.comNathalie Pham | +33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik Elgoutni | +1 617 710 3587 | tarik.elgoutni@sanofi.comThibaud 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 statementsThis 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 regarding the marketing and other potential of the product, or regarding potential future revenues from the product. 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, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, 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. Attachment Press_Release

Dec 11, 2025 CAMBRIDGE, Mass. --(BUSINESS WIRE)--Dec. 11, 2025-- Alnylam Pharmaceuticals, Inc. (“Alnylam”) (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has entered into separate, privately negotiated repurchase agreements with certain holders of its 1.00% Convertible Senior Notes due 2027 (the “Notes”) to repurchase for cash (the “Repurchases”) approximately $34.4 million aggregate principal amount of the Notes for a total repurchase cost (including accrued and unpaid interest) of approximately $51.9 million . The final aggregate cash repurchase price is subject to adjustment as a portion of the repurchase price will be based in part on the daily volume-weighted average price per share of the Company’s common stock over an agreed measurement period beginning on, and including, December 11, 2025 . The Repurchases are expected to close shortly after completion of the measurement period, subject to the satisfaction of customary closing conditions. Following such closings, approximately $362.8 million aggregate principal amount of the Notes will remain outstanding. The Company had previously entered into capped call transactions with certain financial institutions in connection with the issuance of the Notes. All of these transactions are expected to remain in effect notwithstanding the Repurchases. This news release shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. This news release shall not constitute an offer to purchase, or a redemption notice for, any of the Company’s outstanding Notes. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products include AMVUTTRA® (vutrisiran), ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran), which are being developed and commercialized by Alnylam , and Leqvio® (inclisiran) and Qfitlia™ (fitusiran), which are being developed and commercialized by Alnylam’s partners, Novartis and Sanofi, respectively. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Forward-looking Statements Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, statements regarding: the Repurchases, the amount of Notes that will remain outstanding after the Repurchases and the capped call transactions, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P5x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC . In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. View source version on businesswire.com: https://www.businesswire.com/news/home/20251211966876/en/ Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) +1-617-682-4340 Josh Brodsky (Investors) +1-617-551-8276 Source: Alnylam Pharmaceuticals, Inc.