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The Food and Drug Administration has granted priority review to Roche’s emicizumab-kxwh (Hemlibra) for the treatment of adults and children with hemophilia A without factor VIII inhibitors.

The agency is scheduled to make a decision on approval in October 2018.

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Wikimedia Commons/FitzColinGerald/Creative Commons License
The decision to grant priority review status, which allows for an accelerated review, is based on promising early results from the HAVEN 3 trial, which were recently presented at the World Federation of Hemophilia 2018 World Congress.

Among patients aged 12 years and older without factor VIII inhibitors, emicizumab-kxwh prophylaxis every week reduced treated bleeds by 96% (P less than .0001) and treated bleeds were reduced by 97% (P less than .0001) in patients who were treated every 2 weeks, according to Roche. The drug-treated group was compared with patients who received no prophylaxis. Another arm of the study examined patients who had previously received factor VIII prophylaxis and then switched to emicizumab-kxwh prophylaxis. In an intrapatient comparison, emicizumab-kxwh showed a 68% reduction in treated bleeds, which was statistically significant and demonstrated superior efficacy to factor VIII prophylaxis.

Emicizumab-kxwh was approved by FDA in November 2017 for routine prophylaxis for adults and children with hemophilia A with factor VIII inhibitors. That approval was based on results from the HAVEN 1 and HAVEN 2 studies.

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The Food and Drug Administration has granted priority review to Roche’s emicizumab-kxwh (Hemlibra) for the treatment of adults and children with hemophilia A without factor VIII inhibitors.

The agency is scheduled to make a decision on approval in October 2018.

FDA icon
Wikimedia Commons/FitzColinGerald/Creative Commons License
The decision to grant priority review status, which allows for an accelerated review, is based on promising early results from the HAVEN 3 trial, which were recently presented at the World Federation of Hemophilia 2018 World Congress.

Among patients aged 12 years and older without factor VIII inhibitors, emicizumab-kxwh prophylaxis every week reduced treated bleeds by 96% (P less than .0001) and treated bleeds were reduced by 97% (P less than .0001) in patients who were treated every 2 weeks, according to Roche. The drug-treated group was compared with patients who received no prophylaxis. Another arm of the study examined patients who had previously received factor VIII prophylaxis and then switched to emicizumab-kxwh prophylaxis. In an intrapatient comparison, emicizumab-kxwh showed a 68% reduction in treated bleeds, which was statistically significant and demonstrated superior efficacy to factor VIII prophylaxis.

Emicizumab-kxwh was approved by FDA in November 2017 for routine prophylaxis for adults and children with hemophilia A with factor VIII inhibitors. That approval was based on results from the HAVEN 1 and HAVEN 2 studies.

 

The Food and Drug Administration has granted priority review to Roche’s emicizumab-kxwh (Hemlibra) for the treatment of adults and children with hemophilia A without factor VIII inhibitors.

The agency is scheduled to make a decision on approval in October 2018.

FDA icon
Wikimedia Commons/FitzColinGerald/Creative Commons License
The decision to grant priority review status, which allows for an accelerated review, is based on promising early results from the HAVEN 3 trial, which were recently presented at the World Federation of Hemophilia 2018 World Congress.

Among patients aged 12 years and older without factor VIII inhibitors, emicizumab-kxwh prophylaxis every week reduced treated bleeds by 96% (P less than .0001) and treated bleeds were reduced by 97% (P less than .0001) in patients who were treated every 2 weeks, according to Roche. The drug-treated group was compared with patients who received no prophylaxis. Another arm of the study examined patients who had previously received factor VIII prophylaxis and then switched to emicizumab-kxwh prophylaxis. In an intrapatient comparison, emicizumab-kxwh showed a 68% reduction in treated bleeds, which was statistically significant and demonstrated superior efficacy to factor VIII prophylaxis.

Emicizumab-kxwh was approved by FDA in November 2017 for routine prophylaxis for adults and children with hemophilia A with factor VIII inhibitors. That approval was based on results from the HAVEN 1 and HAVEN 2 studies.

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