Emapalumab-LZSG

▎药明康德内容团队编辑 Sobi公司今日宣布，美国FDA已接受为Gamifant（emapalumab）递交的补充生物制品许可申请（sBLA），用于治疗斯蒂尔病（Still’s disease）成人和儿童患者中的噬血细胞性淋巴组织细胞增生症（HLH）/巨噬细胞活化综合征（MAS），这些患者对糖皮质激素应答不足、不耐受或为出现MAS复发。FDA同时授予该申请优先审评资格，预计在2025年6月27日之前完成审评。 HLH/MAS是HLH的一种形式，是风湿性疾病的一种严重并发症，最常见于斯蒂尔病。HLH/MAS是一种罕见的全身性高炎症状态，其常见临床表现包括持续高热、高铁蛋白血症、细胞减少、凝血功能障碍及肝脾肿大。 该申请基于两项研究EMERALD和NI-0501-06的合并数据分析，共纳入39例患者。53%的患者在第8周达到完全缓解，85%的患者在研究期间曾达到完全缓解。治疗2周后，患者平均每周糖皮质激素使用剂量减少了70.1%。 Gamifant是一种单克隆抗体，能够结合并中和干扰素γ（IFN-γ），2018年它获得FDA批准用于治疗对常规HLH治疗耐药或不耐受，出现复发或进展性疾病的成人及儿科原发性HLH患者。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] US FDA grants Priority Review to Sobi's supplemental Biologics Licence Application (sBLA) for Gamifant® (emapalumab-lzsg). Retrieved February 27, 2025, from https://www.prnewswire.com/news-releases/us-fda-grants-priority-review-to-sobis-supplemental-biologics-licence-application-sbla-for-gamifant-emapalumab-lzsg-302387090.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

STOCKHOLM, Feb. 27, 2025 /PRNewswire/ -- Sobi® (STO: SOBI), today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-Izsg) for use in adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. The application was granted Priority Review with a PDUFA date of June 27, 2025. HLH/MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still's disease including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). HLH/MAS is a rare systemic disorder of hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. "HLH/MAS in Still's disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure," said Lydia Abad-Franch, MD, MBA, Head of R&D and Chief Medical Officer at Sobi. "There is no approved therapy for HLH/MAS today. Gamifant® (emapalumab-Izsg) selectively neutralizes interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients." The application is based on results from pooled data from two studies which enrolled a total of 39 patients, the EMERALD (NCT05001737) and the NI-0501-06 (NCT03311854) studies. Fifty-three percent of patients had a complete response at Week 8 and 85% had a complete response at any time during the studies. Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. Gamifant® (emapalumab-Izsg) a monoclonal antibody that binds and neutralises interferon gamma (IFN-y), was approved by the FDA in 2018 for the treatment of adult and paediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinemia, possibly progressing to multiple organ failure and death. MAS is classified as a form of hemophagocytic lymphohistiocytosis (HLH). About Gamifant® (emapalumab-lzsg) Gamifant® (emapalumab-lzsg) is an anti-interferon gamma (IFN-γ) monoclonal antibody that binds to and neutralises IFN-γ. In the USA, Gamifant® (emapalumab-lzsg) is the first and only treatment approved by the FDA for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant® (emapalumab-lzsg) is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2024, revenue amounted to SEK 26 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. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED