2014年11月18日讯 /生物谷BIOON/ --上周五，美国FDA更改了其早前的决定，决定批准制药巨头赛诺菲公司开发的多发性硬化症药物Lemtrada上市。这一决定也表明赛诺菲公司在这一领域再下一城。不过，和其他多发性硬化症药物类似，Lemtrada上市时，也会着重标明这种药物可能会导致自身免疫反应以及其他严重的不良反应。同时药物的使用有增大患者患甲状腺癌等恶性癌症的风险。
大约一年前，FDA就因为担忧Lemtrada的副作用而选择将其拒之门外。然而，经过一年的充足准备，赛诺菲公司向FDA提交了充分的额外数据，令审核人员相信相较于Lemtrada的副作用，其药效更为突出。事实上，即使在FDA下属的专家委员会上，对于Lemtrada是否能够上市也曾存在一些争议。不过最终FDA决定采用批准上市，但严格标示其副作用并控制其适用范围这种折衷的方案。
多发性硬化症一直是各大医药公司竞争的热点，而这一市场也在不断扩大之中。根据调查报告显示，美国、日本、欧盟五大主要市场（法国、德国、意大利、西班牙、英国）多发性硬化症（MS）药物销售以4%的年度复合增长率（CAGR）增长，并在2023年达到200亿美元。因此，罗氏、辉瑞、默克等生物医药巨头都在这一领域部署了自己的产品。根据赛诺菲公司透露的消息，Lemtrada的上市价格要高于默克的同类产品Rebif，定价为15.8万美元。
而FDA的这一决定对赛诺菲也有重要意义。Lemtrada此前是由麻省坎布里奇市的Genzyme公司持有。赛诺菲早前以200亿美元的价格收购了Genzyme公司，Lemtrada就是促使赛诺菲决定这一投资案的重要原因之一。随着Lemtrada的上市，MS医药市场必将又掀起一番新的龙争虎斗。
详细英文报道：
Late on Friday the FDA put out the word that it has reversed its earlier decision against Sanofi's multiple sclerosis drug Lemtrada, agreeing to allow the pharma giant the right to market the treatment-with some tight controls on just who will get this drug for the relapsing form of the disease.
Like other MS drugs on the market, Lemtrada will come with a boxed warning. Theirs includes cautions against "autoimmune conditions, serious and life-threatening infusion reactions and also noting Lemtrada may cause an increased risk of malignancies including thyroid cancer, melanoma and lymphoproliferative disorders." Access to the therapy will be controlled through a restricted distribution program.
And company officials tell reporters that the drug will be priced at a premium to the rival drug Rebif. Lamtrada will be sold for $158,000 for a two-treatment regimen, compared to about $135,000 for a similar course for Rebif from Merck KGaA.
Instead of challenging the FDA's rejection of Lemtrada--a monoclonal antibody--close to a year ago, Sanofi execs opted to present new analysis to deal with regulators' concerns about the trial design. The company handed over additional information on the studies and Sanofi's Genzyme division has since also delivered more efficacy data from studies of the treatment, which may help negotiations with payers.
But Sanofi faced an uphill climb at the FDA. An internal FDA review highlighted the data on an increased risk of autoimmune diseases, serious infections and treatment-emergent malignancies among the patients taking the drug in Sanofi's studies. To be approvable, the FDA initially ruled, Lemtrada would have to demonstrate substantial clinical benefits or the drug's "serious and potentially fatal safety issues" would scuttle the marketing application. A panel review followed with a mixed vote, with a majority saying the safety issues didn't preclude an approval but that Sanofi's clinical trials were inadequate.
The win now marks a major turnaround for Sanofi, which was embarrassed by the setback at the FDA. Lemtrada was one of the key reasons why CEO Chris Viehbacher engineered the $20 billion acquisition of Cambridge, MA-based Genzyme. Its rejection had left a lingering black eye. But Lemtrada can now be sold alongside Aubagio in the U.S., which won an approval about two years ago.
The MS market is huge and fast changing. Biogen Idec has had some signal accomplishments, quickly ramping up sales of Plegridy (tecfidera). Teva, meanwhile, relies on Copaxone for much of its revenue but faces the loss of patent protection.
Viehbacher, though, won't be around the join the celebrations at Sanofi. The board in Paris ousted the CEO weeks ago after growing disenchanted with his management style. But David Meeker, the Genzyme veteran who stayed on to run the subsidiary, is still in place to toast their success.
"Today's approval is the culmination of more than a decade of work by Genzyme to develop Lemtrada," said Meeker. "Lemtrada demonstrated superior efficacy over Rebif on annualized relapse rates in the two studies which were the basis for approval. A comprehensive risk evaluation and mitigation strategy (REMS) will be instituted in order to help detect and manage the serious risks identified with treatment."