/PRNewswire/ -- Sobi® (STO: SOBI) today announced that the U.S. Food and Drug Administration (FDA) approved Gamifant

/PRNewswire/ -- Sobi®（STO：SOBI）今天宣布，美国食品和药物管理局（FDA）已批准Gamifant

(emapalumab-lzsg) for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS..

(emapalumab-lzsg) 用于治疗已知或疑似 Still 病（包括系统性幼年特发性关节炎 [sJIA]）相关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) 的成人和儿童（新生儿及以上）患者，这些患者对糖皮质激素反应不足或不耐受，或出现复发性 MAS。

'With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes,' said

“凭借我们在原发性噬血细胞性淋巴组织细胞增多症方面的专业知识，我们了解快速管理MAS以改善患者预后的紧迫性，”

Guido Oelkers

吉多·奥尔克斯

, Chief Executive Officer at Sobi. 'Gamifant is already an established therapy making a meaningful difference for patients with primary HLH, and with this approval, we are excited about the opportunity to positively impact patients affected by MAS in Still's Disease'.

，Sobi首席执行官。“Gamifant已经成为一种成熟的疗法，为原发性HLH患者带来了显著的改善，随着此次获批，我们很高兴有机会对Still病中受MAS影响的患者产生积极影响。”

The approval is based on results of the pooled data from two pivotal studies, the Phase 3 study (

该批准基于两项关键研究的汇总数据结果，即III期研究 (

NCT05001737

NCT05001737

) and NI-0501-06 (

) 和 NI-0501-06 (

NCT03311854

NCT03311854

). 54 percent (21/39) of patients had a complete response at Week 8, and 82% (32/39) achieved clinical MAS remission (VAS ≤1 cm) at Week 8. Safety and tolerability were consistent with previous clinical studies. In patients with HLH/MAS in Still's disease, the most common adverse events (≥20%) were viral infections, including cytomegalovirus infection or reactivation, and rash..

54%（21/39）的患者在第8周时完全缓解，82%（32/39）的患者在第8周达到了临床MAS缓解（VAS ≤1 cm）。安全性和耐受性与之前的临床研究一致。在患有HLH/MAS的Still病患者中，最常见的不良事件（≥20%）是病毒感染，包括巨细胞病毒感染或再激活以及皮疹。

'MAS in Still's disease is a serious and potentially life-threatening complication, marked by severe hyperinflammation and, in some cases, multi-organ failure,' said

“MAS 在 Still 病中是一种严重且可能危及生命的并发症，其特征是严重的过度炎症反应，并在某些情况下导致多器官衰竭，”

Alexei A. Grom

阿列克谢·A·格罗姆

, MD, Professor of Pediatrics, Research Director Division of Rheumatology, Cincinnati Children's Hospital Medical Center. 'Many patients affected by MAS—both young children and adults—face significant unmet medical needs. With Gamifant now as the first FDA-approved treatment for MAS, we have a new therapeutic option that helps control hyperinflammation and reduce our reliance on high-dose glucocorticoids.'.

医学博士，儿科教授，辛辛那提儿童医院医疗中心风湿病科研究主任。 “许多受 MAS 影响的患者——包括年幼的儿童和成人——面临着重大的未满足医疗需求。随着 Gamifant 现在成为首个获 FDA 批准的 MAS 治疗药物，我们有了一个新的治疗选择，可以帮助控制过度炎症并减少我们对高剂量糖皮质激素的依赖。”

MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still's disease including systemic juvenile idiopathic arthritis and adult-onset Still's disease. HLH/MAS is a rare systemic disorder of interferon gamma (IFNy) driven hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly..

MAS是HLH的一种形式，是风湿性疾病的一种严重并发症，最常发生在Still病中，包括全身型幼年特发性关节炎和成人Still病。HLH/MAS是一种罕见的系统性干扰素γ（IFNy）驱动的高炎症性疾病，常见的临床表现包括持续高热、铁蛋白升高、细胞减少、凝血功能障碍以及肝脾肿大。

Gamifant, an interferon gamma (IFNγ)–blocking antibody, is the first and only FDA approved treatment for adult and pediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.

Gamifant是一种干扰素γ（IFNγ）阻断抗体，是首个也是唯一获得FDA批准的治疗成人和儿童（新生儿及以上）原发性HLH患者的产品，适用于对传统HLH疗法产生难治、复发、疾病进展或不耐受的患者。

About macrophage activation syndrome (MAS)

关于巨噬细胞活化综合征 (MAS)

Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently in Still's disease including systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis.

巨噬细胞活化综合征 (MAS) 是风湿病的严重并发症，最常见于斯蒂尔病，包括全身性幼年特发性关节炎 (sJIA) —— 一种罕见的系统性自身炎症性疾病，常见临床表现包括每日高烧、典型的短暂性皮疹、关节炎、淋巴结肿大、肝脾肿大和浆膜炎。

MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH)..

MAS 的特点是发烧、肝脾肿大、肝功能障碍、血细胞减少、凝血异常和高铁蛋白血症，可能发展为多器官衰竭和死亡。MAS 被归类为继发性噬血细胞性淋巴组织细胞增多症 (HLH)。

About Gamifant® (emapalumab-lzsg)

关于Gamifant®（emapalumab-lzsg）

Gamifant® (emapalumab-lzsg) is the only approved anti-interferon gamma (IFNγ) monoclonal antibody. Gamifant works by binding to and neutralizing IFNγ. When IFNγ is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour..

Gamifant®（emapalumab-lzsg）是唯一获批的抗干扰素γ（IFNγ）单克隆抗体。Gamifant通过结合并中和IFNγ起作用。当IFNγ以不受控制的方式分泌时，体内会发生过度炎症。Gamifant适用于通过静脉输注一小时给药。

Gamifant is approved in the US for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.

Gamifant在美国被批准用于治疗成人和儿童（新生儿及以上）原发性噬血细胞性淋巴组织细胞增多症（HLH）患者，适用于对常规HLH疗法产生耐药性、复发性或进展性疾病或不耐受的患者。

Gamifant is also approved in the US for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic 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..

Gamifant还被美国批准用于治疗已知或疑似Still病的成人和儿童（新生儿及以上）患者，这些患者患有噬血细胞性淋巴组织细胞增多症（HLH）/巨噬细胞活化综合征（MAS），对糖皮质激素反应不足或不耐受，或出现复发性MAS。

About Sobi®

关于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 1,900 employees across

Sobi是一家全球生物制药公司，致力于释放突破性创新的潜力，改变罕见病患者的日常生活。Sobi在全球拥有约1,900名员工，分布在各地。