CFDA药化司司长王立丰在 7月4日 上午的新闻发布会上介绍，我国在研的预防手足口病EV71灭活(Vero细胞)疫苗已经陆续完成临床试验，进入生产注册审评阶段，目前已进入审评的最后阶段，很快就会上市。“如果现在出来，将会成为全球第一个EV71疫苗。”
手足口病是一种儿童传染病，多发生于5岁以下儿童，可引起手、足、口腔等部位的疱疹，少数患儿可引起心肌炎、肺水肿、无菌性脑膜脑炎等并发症，个别重症患儿如果病情发展快，将导致死亡。手足口病常由肠道病毒EV71感染引起，目前国内外尚未有相应疫苗上市。
手足口病疫苗属于国内需求急迫、市场潜力较大的品种之一，目前涉足研发的企业不在少数。研发领先者分别为国内的微谷生物医药（属中生股份）、科兴生物(NASDAQ:SVA)、中国医学科学院医学生物学研究所（昆明所），三者已相继申请了该疫苗的生产批件；科兴生物、中生股份甚至在去年下半年就开始着手准备配套生产车间及GMP认证工作。
此外，华兰生物也在去年2月申报了吸附手足口病(EV71型)灭活疫苗(Vero细胞)临床。
我国成功研制先进回旋加速器 可用于生产治疗癌症同位素药物
【我国研制成功先进回旋加速器】国防科工局今天宣布，我国在核物理研究领域取得重大进展，由中核集团原子能科学研究院完成的回旋加速器首次调试出速度接近一半光速的质子束。回旋加速器可生产治疗癌症同位素药物，其产生的电子束甚至能用于清除草莓上的细菌。
安进白血病试验药物Blinatumomab获FDA突破性治疗药物资格 发布日期：2014-07-05 来源：fiercebiotech
近日，安进旗下白血病试验药物Blinatumomab赢得了FDA突破性治疗药物资格。
近日，安进旗下白血病试验药物Blinatumomab赢得了FDA突破性治疗药物资格。该资格的获得是基于其在费城阴性复发或难治性早期B细胞型急性淋巴细胞性白血病（ALL）2期试验中的出色数据，这种疾病是一种致命的血液及骨髓癌症。这一资格的授予使安进朝着实施NDA迈进了一大步。在获得突破性治疗药物资格之际，安进的研发团队正面临越来越大的压力。现在，安进可以宣传Blinatumomab的近期前景，并将其归入关键治疗药物如T-Vec和PCSK9药物Evolocumab (AMG 145) 的行列。这款药物是一种双特异性T细胞单链抗体(BiTE)，简单说，它利用一种抗体可以重新定向T细胞杀手以毁灭肿瘤细胞。新型免疫肿瘤治疗药物可刺激免疫系统对癌症进行攻击，这类药物已成为癌症领域的一个热点，也吸引了众多制药巨头的大量投资。默沙东目前的研发主管Perlmutter在以12亿美元收购生物技术公司Micromet时，显然已迷恋上了这类药物。安进一位发言人称，2期研究达到了主要终点，在经过Blinatumomab 两轮治疗后，43%的患者获得了完全缓解或部分血液学复苏的完全缓解。数据已在ASCO上进行了发布。“治疗复发和难治性ALL患者的新药有很高的未满足的医疗需求，这类患者很少有治疗选择，” Harper在一份声明中称。“在复发或难治性ALL患者身上评价Blinatumomab的2期试验结果是令人鼓舞的，为这位药物的获批提供了坚实的基础。”信源地址：http://www.fiercebiotech.com/sto ... rough-title-leukemia-drug/2014-07-01
FDA初步批准Spectrum治疗淋巴瘤药物Beleodaq上市
美国FDA最近宣布初步批准由Spectrum生物医药公司开发的用于治疗T细胞淋巴瘤药物belinostat上市，这种药物将以Beleodaq的商品名出售。外周T细胞淋巴瘤是一种罕见并发展迅速的非霍奇金淋巴瘤淋巴瘤。这种疾病每年会杀死18990名患者。此次批准的Beleodaq是一种组蛋白脱乙酰酶抑制剂。也是自2009年至今批准的第三种治疗这种疾病的药物。这一药物的批准也意味着Spectrum公司获得了BioAlliance Pharma公司的里程碑基金。根据一项有129名患者参与的临床三期研究，使用Beleodaq的患者有25.8%病情得到改善。而患者常见的副作用主要是贫血、疲劳、发热、恶心呕吐等。详细英文报道：Spectrum Pharmaceuticals ($SPPI) has won an early nod from the FDA for belinostat, a new T-cell lymphoma drug that will be sold as Beleodaq.Peripheral T-cell lymphoma is a rare and fast-growing type of non-Hodgkin lymphoma characterized by cancerous lymph nodes. The disease accounts for about 1 in 10 of the 70,800 new cases of NHL reported each year, which kills 18,990 of them.The approval for the drug, a histone deacetylase inhibitor (HDAC), comes more than a month ahead of the August 9 PDUFA date."This is the third drug that has been approved since 2009 for the treatment of peripheral T-cell lymphoma," said Richard Pazdur, M.D., director of the Office of Hematology and oncology Products in the FDA's Center for Drug evaluation and Research. "Today's approval expands the number of treatment options available to patients with serious and life-threatening diseases."The approval for Spectrum, which in-licensed the U.S. rights for this drug, is a boon for Denmark's Topotarget, which developed the drug. The biotech is now in line for a $25 million milestone as it preps for a merger with BioAlliance Pharma. Back in 2010, when Spectrum inked the deal, it paid $30 million upfront and agreed to pay up to $320 million in milestones, along with a million shares of stock and royalties on sales. Spectrum got the rights to North America and India along with an option on China.The safety and effectiveness of Beleodaq was evaluated in a clinical study involving 129 participants with relapsed or refractory PTCL, according to the FDA. All patients were treated with Beleodaq until their disease progressed or side effects became unacceptable. "Results showed 25.8% of participants had their cancer disappear (complete response) or shrink (partial response) after treatment. The most common side effects seen in Beleodaq-treated participants were nausea, fatigue, fever (pyrexia), low red blood cells (anemia), and vomiting.""We are very pleased with the validation of our compound and find that it truly underlines the rationale behind Topotarget's merger with BioAlliance Pharma in providing an even stronger orphan oncology pipeline for the combined entity, Onxeo," Anders Vadsholt, CEO of Topotarget, said in a statement.
FDA承诺对Boehringer肺部疾病药物nintedanib优先审核
FDA最近表示将对勃林格殷格翰公司开发的用于治疗肺部疾病特发性肺纤维化（idiopathic pulmonary fibrosis (IPF)）药物nintedanib进行优先审核。这一决定也将加快其上市的审批流程并在与InterMune公司相似药物竞争中迎头赶上。勃林格殷格翰公司进行了两项超过1000名患者参与的临床上拿起研究，分别发现nintedanib将患者肺功能年平均丧失率降低了48%和55%，而对照组仅为5%。在美国目前还没有一种治疗IPF的特效药物，据了解，美国每年有40000人死于该病。而勃林格殷格翰公司的竞争对手InterMune公司开发的类似产品Pirfenidone则已经在今年五月份提交FDA审核并预计在六个月内获得批准。详细英文报道：The FDA is guaranteeing a priority review for Boehringer Ingelheim's much-watched treatment for a rare lung disease, likely speeding up the drug's path to market as the German pharma races with biotech InterMune ($ITMN).Boehringer's treatment, dubbed nintedanib, is a therapy for idiopathic pulmonary fibrosis (IPF), an often fatal disease that scars the lungs and stands in the way of oxygen absorption. In two Phase III studies on more than 1,000 patients, the drug reduced patients' annual rate of lung-function decline by 48% and 55%, compared to just 5% with placebo arm.In the U.S., there are no approved treatments for IPF, which kills about 40,000 people a year, according to the Coalition for Pulmonary Fibrosis. The FDA's priority review designation for nintedanib will give Boehringer wider access to regulators and a faster path to a final decision, under agency rules, a regulatory victory that should come in handy as the company strives to hit the U.S. market before an encroaching rival.Pirfenidone, InterMune's competing IPF drug, endured an FDA rejection in 2010, but, thanks to promising new Phase III studies, the biotech has worked its way back into the good graces of investors. InterMune resubmitted the treatment in May and expects to win approval within 6 months, launching pirfenidone in the first quarter of 2015 if all goes according to plan.Boehringer isn't disclosing its planned timeline for nintedanib, but even if InterMune wins the stateside race, the company has to like its commercial chances. Pirfenidone is already on the market in Europe, but, despite two years of momentum, the drug brought in just $70.2 million last year. The European Medicines Agency last month agreed to an accelerated review of nintedanib, and Boehringer, with its commercial heft, should be able to compete if and when it launches nintedanib on the continent.Analysts have said the U.S. market for IPF treatments could peak at north of $2 billion, but just which company will claim the largest share of it remains up for debate. InterMune's shares shot up in March after Boehringer revealed that nintedanib missed a secondary endpoint in one of its Phase III trials, but a May editorial in The New England Journal of Medicine heralded the drug's efficacy and potential as a new hope for IPF patients.
Jazz继续开发治疗静脉闭塞症药物defibrotide
Jazz生物医药公司在去年收购了著名罕见病药物研究公司Gentium公司后，立刻开始了一系列动作。现在，Jazz公司开始将目光投向一项此前被Gentium公司长期搁置的治疗静脉闭塞症药物defibrotide。此前Gentium公司与西格玛公司曾经达成一项总价值2500万美元的合作协议以研发defibrotide。然而在2013年欧洲医药管理部门拒绝了其上市申请并列举出defibrotide从药效到安全性一系列问题，给公司以沉重打击。另一方面，2011年FDA也曾作出类似决定。此后由于Gentium寻求重组，defibrotide的开发审批工作就陷入暂停状态。此次Jazz公司决定继续这一项目，显然是有把握推动相关医药管理部门批准这一药物。详细英文报道：When Jazz Pharmaceuticals ($JAZZ) bought out the rare-disease drug developer Gentium ($GENT) for a billion dollars late last year, the company picked up a new therapy for severe veno-occlusive disease that it went on to launch in Europe last April. Now it's buying back the remaining U.S. rights to the drug--defibrotide, which Gentium had bargained away to Sigma-Tau--for $75 million in cash and up to $175 million in milestones. And that extra cash will be staked entirely on its ability to shove the long-delayed drug through the FDA approval process.Defibrotide has traveled a long and rocky road. Back in early 2013 Gentium's shares went into a nosedive after the company revealed that regulators in the EU had raised a host of issues on the antithrombotic drug. The Italian company had already been stiff-armed by the FDA way back in 2011, when regulators raised some thorny questions about the "completeness of the datasets for both the treatment and prevention studies. The FDA requested that the company conduct additional quality reviews of the original datasets and databases. The FDA also requested additional details regarding the conduct and monitoring of the trials by the independent review committee."As a result, Gentium yanked its application and said it would regroup.Jazz is now betting that they can get the FDA to approve the therapy, and Sigma-Tau's milestones are pegged against the timing of that approval. Jazz will be building its case around trial data that demonstrated an improved survival rate for patients on the drug, though 18% of the patients in the drug arm were forced out of the study due to toxicity."While the low valuation ($75m upfront + milestones) will likely come as a surprise to investors, it is important to remember that JAZZ already owned 40% of the economics on US defibrotide," noted Leerink's Jason Gerberry. "The deal looks relatively break-even assuming ~$210m in peak sales (1.5K pts at $140k per patient/yr), which assumes modest off-label use."There are no approved therapies for severe veno-occlusive disease in the U.S., in which the small veins in the liver are blocked. The absence of therapies prompted the EU's CHMP to reverse its stand on approval a year ago, even though regulators noted that they didn't fully understand the mechanism of action and fretted that the therapy carries some dangerous side effects."The benefits with Defitelio (its trade name) are its effects of defibrotide which increase the breakdown of clots in the blood," the CHMP concluded. "In addition there is experimental evidence that Defibrotide may protect the cells lining blood vessels. The most common side effects are hemorrhage (including but not limited to gastrointestinal hemorrhage, pulmonary hemorrhage and epistaxis), hypotension and coagulopathy.""This transaction would not only strengthen our global presence, but also demonstrate our commitment to diversify and expand our U.S. commercial portfolio with meaningful new therapies," said Russell Cox, executive vice president and chief operating officer of Jazz Pharmaceuticals, in a statement. "We look forward to ongoing discussions with the FDA as we continue our efforts toward submission of an NDA for defibrotide in the U.S. Patients in the U.S. with severe VOD have a critical unmet medical need, and we believe that defibrotide has the potential to become an important treatment option for these patients."
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