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克利夫兰医学研究中心评出三种最具突破性药物
发布时间:[2013/10/25]     访问人数:[1188]

 --著名医学研究机构克利夫兰医学中心都会在最近处于后期研究的药物中选出一些最具突破性的药物。今年这一排名的前三位被Gilead公司的sofosbuvir、诺华公司的心脏病药物serelaxin以及Pharmacyclics公司的抗癌药ibrutinib所占据。

Sofosbuvir是一种用于治疗丙肝的特效药物,这种药物可以通过口服的方式给药,sofosbuvir被认为是能够取代目前常用的注射式的干扰素治疗方法。Serelaxin则是一种人工合成的激素可以用于孕妇使用,而没有明显的副作用。Ibrutinib则是通过激活B细胞介导的免疫系统来抑制癌细胞的扩散。这三种药物都已经获得了FDA突破性药物认证。

今年的这一排名还凸显了小型医药公司的贡献,因为这些上榜药物中有很多是大型药企从小型生物技术公司手中购买所得。

详细英文报道:

Every year the Cleveland Clinic picks out what it sees as the best late-stage development projects in the healthcare industry. And this year it chose to highlight three of the biggest potential blockbusters in the biopharma business.

Ranked in order, the Cleveland Clinic selected Gilead's ($GILD) sofosbuvir, Novartis' ($NVS) heart drug serelaxin and the cancer drug ibrutinib from Pharmacyclics ($PCYC) and J&J ($JNJ) as the three experimental drugs likely to make the biggest near-term impact on people's health.

Sofosbuvir is the first likely entry in a list of new hepatitis C remedies that will offer millions of patients a chance to quell the virus with an oral approach the can eliminate the need for interferon, an injectable which frequently has a harsh effect on patients. Serelaxin takes a page from nature, offering a synthetic version of a hormone known to help with the circulation of blood in pregnant women, offering a new therapy that analysts believe can scoop up blockbuster revenue in the big cardiovascular field. And ibrutinib is the new B-cell receptor pathway inhibitor designed to scramble the division and spread of cancer cells.

All three of these drugs have been in the R&D spotlight for some time now, earning, for example, "breakthrough therapy" designations at the FDA. (For Gilead the "breakthrough" designation was awarded for a promising sofosbuvir combo in the clinic.) The Cleveland Clinic's selection, though, helps highlight what the nation's top docs are looking forward to using in their practices, helping the sponsor companies back up projections of major sales and adding some significant boasting rights to the reputation for cutting-edge innovation.

Perhaps inadvertently, the selection also highlights the role that smaller biotechs play in developing important new products. Novartis picked up serelaxin in a $620 million buyout deal with Corthera back in 2009, while J&J stepped in with one of its trademark $1 billion deals to take a major stake in ibrutinib. Gilead, of course, tops them all with the $11 billion Pharmasset buyout that snagged sofosbuvir.

At the time, some analysts shook their heads over the price for a risky experimental therapy. Now, with peak sales projections going up and up as a likely approval nears, Gilead's deal team is looking prescient.