Boehringer开发抗凝血剂解毒剂获FDA突破性药物认证

德国生物制药巨头Boehringer公司最近开发的用于阻抗其抗凝血药物Pradaxa的解毒剂idarucizumab已经获得FDA的突破性疗法认证。下一步Boehringer公司将抓紧时间将其推上市场。这种解毒剂是一种人源抗体片段，能够特异性阻断Pradaxa的抗凝血作用。目前这种药物已经进入临床研究招募阶段。同时，Boehringer公司还表示这并不是说idarucizumab只对Pradaxa有效，它对现在市面上的许多抗凝血因子类药物都有作用，其中包括了拜耳和强生的Xarelto 以及施贵宝和辉瑞的Eliquis。不过公司也强调这并不是说Pardaxa等抗凝血药物存在安全隐患。Pradaxa 去年销售额达到了17亿美元之多。Idarucizumab仅是用于意外情况下的出血不止问题。

详细英文报道：

German giant Boehringer Ingelheim is racing to the market with an antidote to its blockbuster blood-thinner Pradaxa, securing the FDA's coveted breakthrough therapy designation for its in-development treatment.

The antidote is a fully humanized antibody fragment designed to reverse the anti-coagulant effects of Pradaxa, which prevents clotting by blocking the enzyme thrombin. Dubbed idarucizumab, Boehringer's reversal agent charted immediate, complete and sustained reversal of Pradaxa's effects in a Phase I study, the company said, and a Phase III trial is in the midst of enrollment.

The FDA's breakthrough tag guarantees a speedy review and a front-row seat with regulators, and Boehringer is planning to pursue an accelerated approval for its antidote.

Boehringer says idarucizumab is the first specific antidote for any of the new class of oral anticoagulants--which includes Bayer and Johnson & Johnson's ($JNJ)Xarelto as well as Bristol Myers-Squibb ($BMY) and Pfizer's ($PFE) Eliquis.

Pradaxa sales grew 9% to about $1.7 billion last year, likely creating a sizable market for a treatment to reverse its effects in uncontrolled bleeding situations.

But Boehringer's not alone in the antidote space. Portola Pharmaceuticals' ($PTLA) has a breakthrough tag of its own for andexanet alfa (PRT4445), an anti-anticoagulant already in Phase III trials to reverse the effects of Factor Xa blockers. Portola's drug essentially works as Factor Xa decoy, fooling inhibitors like Xarelto into targeting it instead of the body's coagulation-regulating enzymes and thus halting the effect of blood thinners and keeping patients safe in major bleeding events.

Boehringer is quick to point out that the development of an antidote in no way undermines its faith in Pradaxa's safety. In May, the company agreed to pay about $650 million to settle more than 4,000 lawsuits tied to the blood thinner, a move that came just months after the FDA reviewed data on the drug and concluded that, when used as directed, it's as safe as warfarin.