Avatrombopag Maleate

Sobi's presentations highlight research across multiple rare diseases in haematology including PNH, ITP, HLH and myelofibrosis. STOCKHOLM, June 11, 2026 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new clinical and real-world data will be presented at the 2026 Congress of the European Hematology Association (EHA2026), taking place June 11–14 in Stockholm, Sweden. The presentations include data across paroxysmal nocturnal haemoglobinuria (PNH), immune thrombocytopenia (ITP), primary haemophagocytic lymphohistiocytosis (HLH), and myelofibrosis. The data include real-world evidence and post hoc analyses for Aspaveli® (pegcetacoplan), Doptelet® (avatrombopag), Gamifant® (emapalumab), and Vonjo® (pacritinib). "As EHA comes to Stockholm this year, we are pleased to present new clinical and real-world evidence spanning multiple rare diseases," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. "These presentations reflect the breadth of our ongoing research across PNH, ITP, HLH and myelofibrosis, and continue to build understanding of treatment outcomes in areas of high unmet need." About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally. It is the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally. About the Sobi® and Apellis (now part of Biogen) collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® (avatrombopag) Doptelet® is an orally administered thrombopoietin receptor agonist (TPO-RA) that increases platelet count. It is approved in the United States, European Union and Japan for thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, and for ITP in adults according to local labeling. In the United States, Doptelet® is also approved for pediatric patients 1 year and older with persistent or chronic ITP who have had an insufficient response to a previous treatment. About Gamifant (emapalumab) Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralising interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour. Gamifant is approved in the US for the treatment of adult and paediatric patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and paediatric patients with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS . About Vonjo (pacritinib) Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 2 000 employees across Europe, North America, the Middle East, Asia and Australia. In 2025, revenue amounted to SEK 28 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. Visiting address Norra Stationsgatan 93A, Stockholm +46 8 697 20 00 [email protected] This information was brought to you by Cision . The following files are available for download: 21% more press release views with Request a Demo

近日，央视曝光呼和浩特一起离谱的天价售药乱象：一款纳入国家集采、市场价仅百元左右的救命药，当地京远大药房竟标价3960元售卖，价格相差近40倍。事件曝光后引发全网热议，国家医保局迅速发布通报，属地监管部门立案核查、启动全市专项整治，撕开了集采政策落地背后暗藏的价格漏洞与监管盲区。 此次涉事药品为马来酸阿伐曲泊帕片，是临床常用的血小板提升救命药，多用于住院重症患者救治，刚需属性极强。该药最初以原研名“苏可欣”上市，上市之初单价接近4000元，价格高昂。随着药品集采改革推进，国内二十余家药企完成仿制药获批，南京正大天晴生产的仿制药“晴安欣”于2023年获批上市。 2026年起，该药正式执行第十一批国家集采新政，集采降幅超98%。根据国家集采规则，10片装规格中选价仅49元至89.99元，彻底告别天价药时代，切实降低了重症患者的就医负担。 而此次宰客事件，恰恰利用了患者的就医刚需与信息不对称。呼和浩特市民张先生住院治疗期间，急需该款救命药，家属情急之下在医院紧邻的京远大药房，花费3960元购买了一盒南京正大天晴药企生产的同款药品。家属事后查询比价发现，同城其他药店同款同规格药品，严格执行集采价仅售89.99元，巨大的价格落差让人瞠目结舌。更讽刺的是，面对记者问询，药店店员以“不知集采降价、未收到调价通知”为由搪塞推脱。但核查药店内部台账证实，药品渠道商早在今年3月就已下发正式调价通知，药店明知集采降价政策，却刻意隐瞒、拒不调价，恶意赚取暴利。最终在舆论介入与协商后，药店才为患者退还全部差价。事件曝光后，监管部门迅速落地处置措施。5月30日，国家医保局发布官方通报，呼和浩特市医保局联合市场监管局成立专项工作组，完成现场核查：对涉事药店主要负责人开展约谈，市场监管部门依法行政立案，同时对相关责任人落实医保支付资格记分惩戒。