Genentech’s Hemlibra Significantly Reduces Bleeding in Late-Stage Hemophilia Studies


Genentech’s hemophilia treatment Hemlibra continues to demonstrate its efficacy in treating patients. New late-stage data presented at World Federation of Hemophilia 2018 World Congress shows the drug significantly reduced treated bleeds in a broad patient population in two separate studies.

In the HAVEN 3 study people with hemophilia A without factor VIII inhibitors who received Hemlibra (emicizumab-kxwh) prophylaxis every week or every two weeks showed a 96 percent reduction and 97 percent reduction in treated bleeds, respectively. That testing was against no prophylaxis. In a subset of patients in the HAVEN 3 study who previously received factor VIII prophylaxis, the standard of care, HEMLIBRA reduced bleeds by 68 percent compared to their prior therapy. Additionally, data from the HAVEN 3 trial showed 55.6 percent of people treated with Hemlibra every week and 60 percent who were treated every two weeks experienced zero treated bleeds, compared to 0 percent of people treated with no prophylaxis.

In April the FDA granted Breakthrough Therapy Designation to Hemlibra for people with hemophilia A without factor VIII inhibitors, based on data from the HAVEN 3 study.

In Genentech’s HAVEN 4 study data showed that dosing with Hemlibra every four weeks provided clinically meaningful control of bleeding in people with or without factor VIII inhibitors. Genentech said the HAVEN 4 results show that administration of Hemlibra once per month can provide clinically meaningful control of bleeding in people with hemophilia A with or without factor VIII inhibitors.

Sandra Horning, Genentech’s chief medical officer and head of Global Product Development, said the data from the two trials show Hemlibra controls bleeds in hemophilia A patients and offers the “flexibility of less frequent subcutaneous dosing options.” That flexibility is a selling point that Genentech is touting. In a previous interview Dr. Gallia Levy, head of Hemlibra research at Genentech, told BioSpace that the less frequent dosing will help improve quality of life for patients. Before Hemlibra was approved, Levy said the hemophilia patients had to receive intravenous infusions every few days.

The data from the HAVEN 3 and HAVEN 4 trials, combined with data from two previous Phase III studies, reinforces the overall efficacy and safety of Hemlibra in the care for people with hemophilia A, Horning said.

Genentech is taking the data from the two latest trials and plans to submit them to regulatory agencies for expanded approval of its hemophilia drug. With expanded approval, analysts predict that Hemlibra could generate up to $5 billion annually for the Roche subsidiary.

Hemlibra was approved in November 2017 by the U.S. Food and Drug Administration. It was the first medication approved in 20 years for Hemophilia A with inhibitors. In December Genentech showed off long-term data from its HAVEN 1 and HAVEN 2 trials at the American Society of Hematology meeting. The longer-term data shows that more patients continued to experience zero bleeds with Hemlibra prophylaxis compared to their prior prophylaxis or on-demand BPA treatment. Updated data from HAVEN 2 showed 94.7 percent of children with hemophilia A with inhibitors who received Hemlibra prophylaxis had zero treated bleeds. Updated data from the HAVEN 1 study showed an 88 percent reduction in treated bleeds in adults and adolescents.

Hemlibra does come with a boxed warning due to potential blood clots in patients. Genentech has included guidelines in dosing protocols to address those concerns.

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