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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for Portola Pharmaceuticals' Factor Xa inhibitor antidote andexanet alfa, which reverses the anticoagulant effect of direct or indirect Factor Xa inhibitors in patients experiencing a serious uncontrolled bleeding event or who require urgent surgery.
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At present, there is no approved antidote to treat these patients and andexanet alfa has the potential to become the first approved universal antidote for Factor Xa inhibitors and the standard of care to manage bleeding associated with these anticoagulants.
Portola chief executive officer Bill Lis said: "Orphan drug designation for andexanet alfa recognizes its potential to address a significant unmet medical need and to advance the field by helping patients who currently have no treatment options.
"Four factor prothrombin complex concentrates (PCCs), which are indicated for warfarin reversal, have not demonstrated reversal of Factor Xa inhibitor activity and have a black box warning for fatal and non-fatal thromboembolic events."
Currently, andexanet alfa is the only compound being evaluated as a reversal agent for Factor Xa inhibitors that directly and specifically corrects anti-Factor Xa activity.
The company is evaluating andexanet alfa in two randomized, placebo-controlled Phase III ANNEXA registration trials using pharmacodynamic endpoints agreed to with the FDA, including anti-Factor Xa inhibitor units, to show efficacy.