Hemlibra Superior to Factor VIII Therapies in Hemophilia A Patients Without Inhibitors, Trial Data Show

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows.

The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial” was published in the journal Current Medical Research and Opinion.

Hemlibra (by Roche) is a humanized monoclonal antibody approved in the U.S. as a routine preventive treatment for adults and children with hemophilia A, with or without inhibitors. Hemlibra is approved in Europe as a preventive treatment for all hemophilia A patients with inhibitors, and for severe hemophilia A patients with or without inhibitors.

It works as a bypassing agent to mimic the activity of factor VIII (FVIII) — the missing clotting factor in hemophilia A patients — helping blood to clot normally. It also reduces blood loss, and prevents damage to joints and tissues from internal bleeding.

Hemlibra has a half-life of nearly 30 days and is administered by injections under the skin (subcutaneously), carrying a lower treatment burden. Due to its characteristics, preventive treatment (prophylaxis) with Hemlibra offers a potential advantage to conventional replacement therapy (intravenous infusions of the missing clotting factor), which is linked to a residual bleed rate and long-term joint problems, the researchers said.

Scientists at Roche and colleagues compared the effectiveness of prophylaxis with Hemlibra to FVIII in patients with hemophilia A without inhibitors.

To make this comparison, they did a pooled analysis (called network meta-analyses) of three trials — the A-LONG (NCT01181128), the LEOPOLD II (NCT01233258) and the SPINART (NCT00623480) — assessing the efficacy of FVIII therapy given preventively and on-demand, and of the HAVEN 3 trial (NCT02847637), which evaluated Hemlibra prophylaxis and FVIII on demand (also called no prophylaxis group).

They also performed a sub-group analysis with data from HAVEN 3 alone, where they compared outcomes in patients using FVIII prophylaxis before starting preventive treatment with Hemlibra.

For their analyses, researchers compared bleed rates among studies and patient groups.

Pooled analysis of the four trials showed Hemlibra prophylaxis to be superior to FVIII either on demand or as prophylaxis. Specifically, the total treated bleed rate was 64% lower with Hemlibra prophylaxis compared to FVIII prophylaxis.

HAVEN 3 data revealed the same tendency. Patients receiving Hemlibra prophylaxis had a lower rate of treated bleeds (a reduction of 52.8% to 62%) compared to those using FVIII prophylaxis.

These results “support the superiority of emicizumab [Hemlibra] prophylaxis over FVIII prophylaxis in patients with hemophilia A without inhibitors,” the study concluded.