——首个也是唯一的口服蛋白酶体抑制剂Ninlaro美国FDA核准
2015年11月20日,美国食品和药品监管局FDA授权批准Ninlaro(ixazomib)胶囊剂与其他两种治疗联用为治疗有多发性骨髓瘤曾接受至少一种以前治疗。
多发性骨髓瘤是血癌的一种形式发生骨髓中发现是感染斗争浆细胞(白血细胞的一种类型) 。这些癌性细胞倍增，产生一种异常蛋白和将其他健康的血细胞推出骨髓。该疾病可能导致免疫系统变弱和致其他骨或肾脏问题。美国国家癌症研究所估计美国今年将有26,850多发性骨髓瘤新病例和11,240例相关死亡。
FDA的药品评价和研究中心中血液学和肿瘤产品室主任Richard Pazdur，M.D.说：“当学习到更多有关多发性骨髓瘤的生物学，我们受到有新途径治疗该级别的鼓舞，” “今天的批准是今年被批准为多发性骨髓瘤的第三个药物和提供患者用一种新口服治疗，它减慢疾病进展当其他治疗已失败。” FDA在今年2月批准Farydak(panobinostat)和这个月早些时候批准Darzalex(daratumumab)。
Ninlaro是一类型抗癌药被称为蛋白体抑制剂和提供阻断来自多发性骨髓瘤细胞酶作用，阻止多发性骨髓瘤生长和生存的能力。Ninlaro是第一个口服蛋白体抑制剂和被批准与被批准与FDA-批准治疗对多发性骨髓瘤另外药物被称为Revlimid(来那度胺[lenalidomide])和地塞米松[dexamethasone](皮质激素的一种类型)联用。.
在722例多发性骨髓瘤复发，或对以前治疗不反应患者一项国际，随机化，双盲临床试验中显示的安全性和疗效。研究参加者接受或Ninlaro与来那度胺和地塞米松或安慰剂加来那度胺和地塞米松联用。服用Ninlaro参加者与用其他方案参加者(14.7个月)比较无疾病恶化生存更长(平均20.6个月)。
Ninlaro的最常见不良副作用是腹泻，便秘，血液血小板计数低(血小板减少)，外周神经病变(来自神经损伤麻木和疼痛，通常在手和足)，恶心，外周水肿(液体在引起肿胀皮肤下)，呕吐和背痛。
FDA对Ninlaro授权优先审评和孤儿药物指定。优先审评状态是授予申请药物，它被批准将对严重情况的治疗中安全性或有效性显著改善。孤儿药物指定提供鼓励例如税收减免，用户收费减免，和孤儿药物的专有，以帮助和鼓励对罕见疾病药物的发展。
Ninlaro是在三种不同剂量包括2.3mg，3毫克和4mg的胶囊用于口服给药的形式提供.
Ninlaro是总部在日本大阪的Takeda Pharmaceuticals上市，Farydak由总部在新泽西East Hanover州 的Novartis Pharmaceuticals上市；Darzalex由宾州Janssen Biotech上市；Revlimid由宾州Celgene Corporation 上市。
Ninlaro
Generic Name: ixazomib
Date of Approval: November 20, 2015
Company: Takeda Pharmaceutical Company
Treatment for: Multiple Myeloma
Ninlaro is a type of cancer drug called a proteasome inhibitor and works by blocking enzymes from multiple myeloma cells, hindering their ability to grow and survive. Ninlaro is the first oral proteasome inhibitor and is approved in combination with another FDA-approved treatment for multiple myeloma called Revlimid (lenalidomide) and dexamethasone (a type of corticosteroid).
The safety and efficacy of Ninlaro were demonstrated in an international, randomized, double-blind clinical trial of 722 patients whose multiple myeloma came back after, or did not respond to, previous treatment. Study participants received either Ninlaro in combination with lenalidomide and dexamethasone or placebo plus lenalidomide and dexamethasone. Those taking Ninlaro lived longer without their disease worsening (average 20.6 months) compared to participants taking the other regimen (14.7 months).
The most common side effects of Ninlaro are diarrhea, constipation, low blood platelet count (thrombocytopenia), peripheral neuropathy (numbness and pain from nerve damage, usually in the hands and feet), nausea, peripheral edema (fluid under the skin causing swelling), vomiting and back pain.
The FDA granted priority review and orphan drug designations for Ninlaro. Priority review status is granted to applications for drugs that, if approved, would be a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan drug designation provides incentives such as tax credits, user fee waivers, and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm473771.htm