阿那白滞素获准用于治疗新生儿期发病的多系统炎症性疾病

圣路易斯(MD Consult)——2013年1月8日，Sobi宣布美国食品药品管理局(FDA)已批准Kineret(阿那白滞素)治疗儿童和成人的新生儿期发生的多系统炎症性疾病(NOMID)。

Kineret是一种重组蛋白质药物，可通过与白介素-1(IL-1)的1型受体结合而阻断IL-1的生物活性。IL-1的1型受体在多种组织和器官中均有表达。这是首个也是唯一一个获得FDA批准的NOMID治疗药物。NOMID是冷吡啉相关周期性综合征(CAPS)的最严重类型。Kineret此前已获准用于缓解成人类风湿性关节炎的症状和体征。
根据Sobi在新闻发布会上的介绍，CAPS是一组由NLRP3常染色体显性突变导致的罕见遗传性自身炎症性疾病。CAPS的特征是无法控制的IL-1过度分泌，后者可导致多种炎症性反应的发生，例如发热、对疼痛敏感、骨骼和软骨破坏，以及急性血浆蛋白反应。当疾病属于中度严重时，通常与发作性、强烈、持续性加重，以及发生进行性听力丧失和继发于淀粉样变性的肾脏衰竭有关。最轻度的CAPS表现为寒冷诱发性发热、皮疹和不适。全球CAPS的发病率估计约为1:1,000,000。

Kinerat采用皮下注射给药。Sobi将会提供配有分级标签的预填充注射器，以便对患儿选用灵活的剂量。

在治疗的最初6个月内，NOMID患者报告的最常见不良事件(发生率＞10%)为注射部位反应、头痛、呕吐、关节痛、发热和鼻咽炎。

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ST LOUIS (MD Consult) - On January 8, 2013, Sobi announced that the US Food and Drug Administration (FDA) has approved Kineret (anakinra) for the treatment of children and adults with neonatal-onset multisystem inflammatory disease (NOMID). Kineret is a recombinant protein drug that blocks the biologic activity of IL-1 by binding to the interleukin-1 type 1 receptor, expressed in a wide variety of tissues and organs. It is the first and only FDA-approved therapy for NOMID, the most severe form of cryopyrin-associated periodic syndromes (CAPS). Kineret was previously approved for the reduction of signs and symptoms of rheumatoid arthritis in adults.

According to the Sobi press release, CAPS are a group of rare inherited autoinflammatory diseases caused by autosomal dominant mutations in the gene NLRP3. CAPS is characterized by uncontrolled overproduction of IL-1, which leads to the occurrence of a number of inflammatory responses such as fever, pain sensitization, bone and cartilage destruction, and acute plasma protein responses. When the disease is of intermediate severity, it is typically associated with episodic, intense, and enduring flares and morbidity, including progressive hearing loss and kidney failure secondary to amyloidosis. The mildest form of the disease presents with cold-induced episodes of fever, rash and malaise. The incidence of CAPS is estimated to be 1:1,000,000 worldwide.

Kinerat is administered by subcutaneous injection. Sobi will provide a prefilled syringe with a graduated label to allow flexible dosing in children.

The most common adverse events reported during the first 6 months of treatment (incidence > 10%) in patients with NOMID were injection-site reactions, headache, vomiting, arthralgia, pyrexia, and nasopharyngitis.