2014年8月5日,，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.

SOLIRIS Rx
Generic Name and Formulations:
Eculizumab 10mg/mL; soln for IV infusion after dilution; preservative-free.

Company:
Alexion Pharmaceuticals, Inc
Indications for SOLIRIS:
Treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. Treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Limitation of use: not for treating Shiga toxin E. coli-related HUS.

Adult:
Give by IV infusion over 35 mins; monitor for ≥1hr after completion. ≥18yrs: PNH: initially 600mg weekly for the first 4 weeks, followed by 900mg for the fifth dose 1 week later, then 900mg every 2 weeks thereafter. aHUS: initially 900mg weekly for the first 4 weeks, followed by 1200mg for the fifth dose 1 week later, then 1200mg every 2 weeks thereafter. Supplemental dosing after PE/PI: see full labeling.

Children:
<18yrs: PNH: not established. aHUS: Give by IV infusion over 1–4hrs via gravity feed, syringe-type pump, or infusion pump; monitor for ≥1hr after completion. 5–<10kg: induction: 300mg weekly for 1 dose; maintenance: 300mg at Week 2, then 300mg every 3 weeks; 10–<20kg: induction: 600mg weekly for 1 dose; maintenance: 300mg at Week 2, then 300mg every 2 weeks; 20–<30kg: induction: 600mg weekly for 2 doses; maintenance: 600mg at Week 3, then 600mg every 2 weeks; 30–<40kg: induction: 600mg weekly for 2 doses; maintenance: 900mg at Week 3, then 900mg every 2 weeks; ≥40kg: induction: 900mg weekly for 4 doses; maintenance: 1200mg at Week 5, then 1200mg every 2 weeks. Supplemental dosing after PE/PI: see full labeling.

Pharmacological Class:
Complement inhibitor.

Contraindications:
Unresolved serious Neisseria meningitidis infection. Individuals not vaccinated against Neisseria meningitidis.

Warnings/Precautions:
Increased risk of meningococcal infection. Give meningococcal vaccine at least 2 weeks prior to treatment. Monitor for early signs of meningococcal infection; evaluate and treat if an infection develops. Discontinue eculizumab if undergoing treatment for meningococcal infections. Administering eculizumab treatment with any other systemic infection (eg, S. pneumoniae, H. influenza). PNH: risk of hemolysis after treatment discontinuation; monitor for at least 8 weeks. aHUS: risk of thrombotic microangiopathy (TMA) after treatment discontinuation; monitor for at least 12 weeks; if TMA occurs, consider reinitiating eculizumab, plasma therapy [plasmapheresis, plasma exchange, or fresh frozen plasma infusion (PE/PI)], or appropriate organ-specific supportive measures. Monitor platelets, serum LDH, and creatinine during and after therapy. Pregnancy (Cat.C). Nursing mothers.

Adverse Reactions:
Headache, nasopharyngitis, back pain, nausea, diarrhea, vomiting, abdominal pain, hypertension, upper respiratory tract infection, anemia, cough, peripheral edema, UTI, pyrexia; meningococcal infection (may be fatal), hypersensitivity reactions.

REMS:
YES

How Supplied:
Single-use vials (30mL)—1