Alexion制药8月1日宣布，欧盟委员会（EC）已授予Soliris（eculizumab，依库珠单抗）治疗重症肌无力（Myasthenia Gravis，MG）的孤儿药地位（ODD）。

重症肌无力（MG）是一种由神经-肌肉接头处传递功能障碍所引起的罕见的自身免疫性疾病，临床主要表现为部分或全身骨骼肌无力和易疲劳，活动后症状加重，经休息后症状减轻。重症肌无力（MG）患者肌肉无力，该病可影响患者的行走能力、说话能力、吞咽能力，在某些情况下还能够影响患者的正常呼吸，并可能导致危及生命的肌无力危象（myasthenic crisis）。补体途径被认为在重症肌无力（MG）的病理生理学中发挥着举足轻重的作用。eculizumab可特异性抑制末端补体途径，被认为具有治疗重症肌无力的潜力。

Soliris是一种首创（first-in-class）的终端补体抑制剂，目前已获批用于阵发性睡眠性血红蛋白尿（PNH）和非典型溶血尿毒症综合征（aHUS）的治疗，这是2种超罕见（ultra-rare）、致衰性且危机生命的疾病，由慢性且不受控的补体激活导致。阵发性睡眠性血红蛋白尿（PNH）系后天获得性的红细胞膜缺陷引起的对补体激活异常敏感的慢性血管内溶血，常睡眠时加重，可伴间歇性血红蛋白尿和全血细胞减少症或反复血栓形成。非典型溶血性尿毒症综合征（aHUS）是一种罕见的遗传性疾病，是一种由补体介导的血栓性微血管病，该病累及多系统，以微血管病性溶血、急性肾衰竭和血小板减少为主要特征。

目前，Soliris尚未获任何国家批准用于重症肌无力（MG）。Alexion正在开展一项多中心、安慰剂对照研究，调查Soliris用于难治性全身型重症肌无力（MG）的治疗。

英文原文：

European Commission Grants Orphan Drug Designation to Soliris® (eculizumab) for the Treatment of Patients with Myasthenia Gravis (MG)

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals (NASDAQ:ALXN) today announced that the European Commission has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation. In patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can ultimately lead to profound and debilitating weakness of various muscle groups throughout the body.

“Patients with MG develop debilitating muscle weakness, impairing their ability to walk, speak clearly, swallow and, in some cases, to breathe normally, which could lead to a life-threatening myasthenic crisis,” said Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D at Alexion. “By specifically inhibiting the terminal complement pathway, which is believed to play a pivotal role in the pathophysiology of MG, we believe that eculizumab has the potential to help patients living with this devastating rare disorder.”

Soliris is a first-in-class terminal complement inhibitor and is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is not approved in any country to treat MG. Alexion is enrolling patients in a multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized MG. More information on this trial is available at www.clinicaltrials.gov under the identifier NCT01997229.

The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product status designation would provide Alexion with certain benefits and incentives in the EU, including a period of market exclusivity if Soliris is ultimately approved for the designated indication.

About Myasthenia Gravis (MG)

Myasthenia Gravis is a rare, debilitating neurologic disorder caused by auto-antibodies that recognize a specific target in the nerve-muscle junction, which results in life-long uncontrolled terminal complement activation causing tissue damage and interference with signalling between nerve and muscle fibers.1,2 Patients with MG initially experience weakness in their ocular (eye) muscles, and the disease typically progresses to the more severe and generalized form to include weakness of head, trunk, limb and respiratory muscles. Symptoms can include drooping eyelid, weakness in the arms and legs, slurred speech, difficulty chewing or swallowing, and difficulty breathing, which could lead to a life-threatening myasthenic crisis.

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007), European Union (2007), Japan (2010) and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is indicated to reduce hemolysis. Soliris is also approved in the U.S. (2011), the European Union (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). Soliris is indicated to inhibit complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). For the breakthrough innovation in complement inhibition, Alexion and Soliris have received the pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.