Ocrevus(ocrelizumab)注射剂第一个药物被批准为原发性进展型多发性硬化另一种治疗选择 2017年3月28日美国食品和药品监管局批准Ocrevus (ocrelizumab)治疗成年患者有复发型多发性硬化症(MS)和原发进展性多发性硬化症(PPMS)。这是第一个被FDA为PPMS批准的药物。Ocrevus是由卫生保健专业人员静脉输注给予。 FDA的药品评价和研究中心的神经学产品部主任Billy Dunn，M.D.说：“多发性硬化对人的生命可能有深刻影响，” “这个治疗不仅为有复发MS患者提供另一种治疗选择，而且首次为那些有原发性进展型多发性硬化患者提供另一种治疗选择。” MS是一种中枢神经系统慢性，炎症，自身免疫疾病，它破坏脑和机体其他部位间交流。它是在年青成年神经失能最常见原因和在妇女比男性更频繁发生。对有MS大多数人，恶化功能的发作(复发)是初始地接着是恢复阶段(缓解)。随着时间的推移，恢复可能是不完全的，导致在功能中进展性下降和增加失能。大多数人20和40岁间经历他们的MS的第一个症状。 PPMS的特征是从症状的开始稳定地恶化功能，往往无早期复发或缓解。美国的疾病控制和预防中心估计约15%是有PPMS的MS患者。 在两项临床试验在1,656例共96周治疗参加者显示Ocrevus 对复发型MS治疗的疗效。两项研究都比较Ocrevus与另一个MS药物。Rebif(干扰素β-1a)。在两项研究中，与Rebif比较患者都接受Ocrevus有减低的复发率和减低的恶化的失能。 在一项PPMS的研究在732被治疗共至少120周参加者，与安慰剂比较接受Ocrevus参加者显示较长时间至残疾失能的恶化。 在有乙型肝炎感染患者或危及生命对Ocrevus输注-相关反应病史不应使用Ocrevus。Ocrevus必须分配与一个患者药物指导描述关于药物的使用和风险的重要信息。Ocrevus可能致输注-相关反应，它可能是严重的。这些反应包括，但不限于，瘙痒的皮肤，皮疹，荨麻疹，皮肤发红，脸红，低血压，发热，疲倦，眩晕，头痛，喉痛刺激，气短，喉头肿胀，恶心，和快速心跳。此外，Ocrevus可能增加对恶性病风险，特别是乳癌。对有活动性感染患者延迟Ocrevus治疗。在接受Ocrevus患者建议不接种活或活减毒疫苗. 除了输注-相关反应，对复发型MS在临床试验中Ocrevus 见到的最常见副作用是上呼吸到感染。在PPMS的研究中最常见的副作用是上呼吸道感染，皮肤感染，和下呼吸道感染。 FDA授权这个申请突破性治疗指定，快速通道指定，和优先审评。 FDA授权Ocrevus批准给予Genentech，有限公司。 推荐剂量：每六个月通过静脉滴注使用。第一剂给予两次300mg输注，间隔两周。随后的剂量作为600mg输注给予。 Ocrevus (ocrelizumab) for Primary Progressive and Relapsing MS Ocrevus (generic name, ocrelizumab) is a humanized monoclonal antibody that was approved by the U.S. Food and Drug Administration on March 28, 2017. Ocrevus was developed by Genentech, a member of the Roche Group, as a treatment for people with multiple sclerosis (MS). The treatment targets B-lymphocytes, a type of immune cell, that express a protein called CD20 on their surface, giving the drug an immunosuppressive function. It is designed to reduce the rates of immune system attacks on myelinated neurons, the main trigger of the disease. Ocrevus is the first FDA-approved therapy that treats both relapsing multiple sclerosis (RRMS) and, in a true breakthrough, primary progressive multiple sclerosis (PPMS), a disease form that previously had no approved treatments. How Ocrevus works Ocrevus, an anti-CD20 monoclonal antibody, targets mature B-cells. Almost 95% of B-cells express the CD20 protein on their surface once they mature and do not shed them, which is what makes CD20 a potent marker for therapeutic purposes. It is believed these CD20-positive B-cells target axons and the myelin sheaths of healthy neurons, initiating a cascading series of immune reactions that lead to MS and disability in patients. Studies have shown that ocrelizumab binds to specific B-cells with CD20 markers, but not to stem cells and plasma cells, preserving vital immune functions within the host. Ocrevus and clinical trials for MS Genentech had announced in February 2016 favorable results from a pivotal Phase 3 trial called ORATORIO, a randomized, double-blind, global and multi center study evaluating the efficacy and safety of ocrelizumab in 732 patients with PPMS. The drug was injected into the bloodstream as two infusions of 300 mg given two weeks apart every six months. At the end of the study period, it was seen that ocrelizumab had met the study’s primary outcome, significantly reducing clinical disease progression in PPMS patients sustained for at least 12 weeks by 24%(compared to placebo), as measured by the Expanded Disability Status Scale (EDSS). That same month, the U.S. Food and Drug Administration (FDA) designated ocrelizumab a Breakthrough Therapy as a possible PPMS treatment, a decision meant to expedite its development and review. Data from two related Phase 3 studies, OPERA I and II, testing the efficacy of the drug in 1,656 patients with relapsing forms of MS, found that ocrelizumab was superior to interferon beta-1a, a well-established MS therapy, reducing the annualized relapse rate (the primary endpoint of both studies) by nearly 50% over a two-year controlled treatment period. Ocrelizumab was into the bloodstream at 600 mg every six months; interferon beta-1a was given by injection under the skin at 44 mcg three times per week. According to Genentech, Ocrevus was the first investigational medicine to show such positive results in patients with both primary progressive and relapsing forms of MS. The company submitted data from all three studies to regulatory authorities in mid-2016, which paved the way for marketing and commercialization of Ocrevus to treat RMS and PPMS. On June 28, 2016, Genentech announced that the company’s Biologics License Application for Ocrevus had been accepted for FDA review, and that the therapy was granted Priority Review, accelerating the review process. All those efforts resulted in the FDA’s decision to approved Ocrevus on March 28, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm549325.htm