武田全球首个口服蛋白酶体抑制剂Ninlaro获欧盟
日本制药巨头武田（Takeda）抗癌管线近日在欧盟监管方面传来喜讯，欧盟委员会（EC）已有条件批准口服蛋白酶体抑制剂Ninlaro（ixazomib），联合Revlimid（lenalidomide，来那度胺）及地塞米松（dexamethasone），用于既往已接受过至少一种治疗方案的多发性骨髓瘤（MM）成人患者。此次批准，使Ninlaro成为欧洲首个也是唯一一个口服蛋白酶体抑制剂。
值得一提的是，Ninlaro在欧洲的监管之路也并非一帆风顺。今年5月底，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）曾发布意见，建议不批准Ninlaro。之后，武田针对这一意见提起上诉，要求CHMP重新审查，最终在今年9月底获得了CHMP支持批准的积极意见。
Ninlaro的获批，是基于关键性III期临床研究TOURMALINE-MM1的积极数据，该研究在复发性和/或难治性多发性骨髓瘤患者中开展，数据显示，与安慰剂+来那度胺+地塞米松联合治疗组相比，Ninlaro+来那度胺+地塞米松联合治疗组无进展生存期（中位PFS：20.6个月vs 14.7个月）显著提高了6个月，达到了研究的主要终点。
此外，在横跨预先定义的各亚组中均观察到了Ninlaro治疗方案的显著疗效。该研究中总生存期（OS）方面的后续分析将在2017年进行。
近年来，尽管临床治疗方面已取得很大的进展，但多发性骨髓瘤仍然是一个棘手的疾病，患者及其治疗医生需要更多的治疗选择。在欧洲，尚没有获批的口服蛋白酶体抑制剂，Ninlaro将填补这一空白。Ninlaro+来那度胺+地塞米松，这种全口服三联疗法将为复发性和/或难治性多发性骨髓瘤（MM）群体提供一种安全有效的、可耐受的口服治疗方案。
在美国，Ninlaro于2015年11月获FDA批准，成为全球上市的首个口服蛋白酶体抑制剂。Ninlaro能够阻断多发性骨髓瘤细胞的酶，进而阻碍其生长及生存的能力。
多发性骨髓瘤（multiple myeloma，MM）是一种浆细胞癌，见于骨髓。多发性骨髓瘤中，一组浆细胞（或骨髓瘤细胞）转化为癌细胞并增生，使浆细胞的数目高于正常水平。由于浆细胞在体内广泛游走，有可能累及体内多数骨骼，可能导致压缩性骨折、骨溶解性病灶和相关疼痛。多发性骨髓瘤可导致若干严重健康问题，累及骨骼、免疫系统、肾脏和个体的红细胞计数，部分较常见症状包括骨骼疼痛和疲乏，疲乏是贫血的症状。多发性骨髓瘤属罕见癌症，每年新发病例在美国约为20，000人、全球约为114，000人。
附：完整资料；
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fcef9088-ebab-4bd8-933f-c35f9c8bd50b
INDICATION AND IMPORTANT SAFETY INFORMATION FOR NINLARO® (ixazomib)
Uses of NINLARO
NINLARO is a prescription medicine used to treat multiple myeloma in combination with the medicines REVLIMID® (lenalidomide) and dexamethasone, in people who have received at least one prior treatment for their multiple myeloma.
It is not known if NINLARO is safe and effective in children.
NINLARO may cause serious side effects, including:
•Low platelet counts (thrombocytopenia) are common with NINLARO and can sometimes be serious. You may need platelet transfusions if your counts are too low. Tell your healthcare provider if you have any signs of low platelet counts, including bleeding and easy bruising.
•Stomach and intestinal (gastrointestinal) problems. Diarrhea, constipation, nausea, and vomiting are common with NINLARO and can sometimes be severe. Call your healthcare provider if you get any of these symptoms and they do not go away during treatment with NINLARO. Your healthcare provider may prescribe medicine to help treat your symptoms.
•Nerve problems are common with NINLARO and may also be severe. Tell your healthcare provider if you get any new or worsening symptoms including: tingling, numbness, pain, a burning feeling in your feet or hands, or weakness in your arms or legs.
•Swelling is common with NINLARO and can sometimes be severe. Tell your healthcare provider if you develop swelling in your arms, hands, legs, ankles, or feet, or if you gain weight from swelling.
•Skin Reactions. Tell your healthcare provider if you get a new or worsening rash.
•Liver problems. Tell your healthcare provider if you get these signs of a liver problem: yellowing of your skin or the whites of your eyes; pain in your right upper-stomach area.
Back pain is also common with NINLARO.
These are not all the possible side effects of NINLARO. Talk to your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Before taking NINLARO, tell your healthcare provider about all your medical conditions, including if:
•You have liver problems or kidney problems or are on dialysis.
•You are pregnant or plan to become pregnant, as NINLARO can harm your unborn baby. Avoid becoming pregnant during treatment with NINLARO. Females, and males with a female partner who is able to become pregnant, must use effective birth control during treatment and for 90 days after the final dose of NINLARO.
•You are breastfeeding or plan to breastfeed. Do not breastfeed during treatment with NINLARO.