Alexion Wins EC Nod for Soliris in Nervous System Disorder

亚力兄罕见病药物Soliris欧盟获批新适应症,治疗视神经脊髓炎谱系疾病

2019-08-29 10:30:00 Zacks

本文共968个字,阅读需3分钟

Alexion Pharmaceuticals, Inc. ALXN announced that the European Commission has approved the label expansion of lead drug, Soliris. The drug is now approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients, who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease. The EC approval was based on comprehensive results from the phase III randomized, double-blind, placebo-controlled PREVENT study and a long-term extension study (ECU-NMO-302), which is still underway. The study met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse. Per the data, 98% of adult AQP4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo at 48 weeks. The encouraging results demonstrated the potential of Soliris to change the treatment paradigm for this rare, devastating, complement-mediated disorder of the central nervous system. We remind investors that the FDA has already approved the drug for the treatment of NMOSD in adult patients, who are AQP4 antibody-positive, in June 2019. A supplemental New Drug Application is currently under review by regulatory authorities in Japan. The drug enjoys an Orphan Drug designation (ODD) for the treatment of NMOSD in the United States, the EU and Japan. Soliris, a first-in-class complement inhibitor that inhibits the C5 protein, is already approved for the treatment of two severe and ultra-rare disorders— paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome — in the United States and Europe. The FDA has also approved the drug for the indication of the generalized myasthenia gravis. The label expansion of the drug should further boost sales. The FDA recently approved Alexion’s long-acting C5 complement inhibitor, Ultomiris, for the treatment of adults with PNH. This consolidated the company’s PNH franchise. It plans to evaluate the drug for the NMOSD indication as well. Shares of the company have gained 13.2% in the year so far against the industry’s decline of 2.3%. We note that Alexion was recently in the news on speculations of a potential buyout by biotech bigwig Amgen AMGN. However, Amgen recently announced an agreement to acquire Celgene Corporation’s CELG plaque psoriasis and psoriatic arthritis drug, Otezla, for more than $13.4 billion, thereby squashing the speculations of buying out Alexion. Alexion’s stock declined on the news. Nevertheless, the company has been one of the prime candidates for acquisition from the onset of 2019 after the mega-merger announcement of Bristol-Myers BMY and Celgene.
亚力兄(Alexion) Pharmaceuticals , Inc . ALXN 公司宣布,欧盟委员会已批准原料药 Solris 的标签扩展。 该药物目前已被批准用于治疗成年患者的视神经脊髓炎频谱障碍( NMOSD ),这些患者是抗水蛋白-4( AQP4)抗体阳性的复发过程的疾病。 EC 的批准是基于 III 期随机、双盲、安慰剂对照的 PRVENT 研究和长期扩展研究( ECU-NMO-302)的综合结果,该研究仍在进行中。研究满足了延长首次判定复发时间和降低复发风险的主要终点。根据数据显示,98%的成年 AQP4抗体阳性患者接受 Solris 治疗后无复发,而在48周时接受安慰剂的患者为63%。令人鼓舞的结果表明,索利斯有可能改变这种罕见的、破坏性的、补体介导的中枢神经系统疾病的治疗模式。 我们提醒投资者, FDA 已于2019年6月批准该药物用于治疗成人患者 NMOSD ,这些患者是 AQP4抗体阳性。日本监管机构目前正在审查一份补充新药申请。该药物享有孤儿药物指定( ODD )的治疗 NMOSD 在美国,欧盟和日本。 Solris 是一种抑制 C5蛋白的一级补体抑制剂,在美国和欧洲已经被批准用于治疗两种严重和极罕见的疾病——阵发性夜间血红蛋白尿症和非典型溶血性尿毒症。FDA 还批准了该药物用于全身重症肌无力的适应症。 药品标签的扩大应该会进一步促进销售。FDA 最近批准亚力兄(Alexion)的长效 C5补体抑制剂 Ultomiris 用于治疗 PNH 成人。这巩固了该公司的 PNH 专营权。它还计划评估 NMOSD 适应症的药物。 该公司股价今年迄今已上涨13.2%,而该行业的跌幅为2.3%。 我们注意到,亚力兄(Alexion)最近在关于生物技术巨头安进(Amgen) AMGN 可能收购的猜测的新闻中。然而,安进(Amgen)最近宣布了一项协议,以134亿美元收购新基(Celgene)公司的 CELG 牌银屑病和银屑病关节炎药物 Otezla ,从而粉碎了收购亚力兄(Alexion)的投机行为。消息传出后,亚力兄(Alexion)的股价下跌。然而,在百时美施贵宝( Bristol-Myers BMY )和新基(Celgene)宣布大规模合并后,该公司一直是2019年初收购的主要候选人之一。

以上中文文本为机器翻译,存在不同程度偏差和错误;偶尔因源网页结构局限,内容无法一次完整呈现。请理解并参考原站原文阅读。

阅读原文