Roche hemophilia drug granted priority FDA review

Evrard Martin
Août 25, 2017

Almost one in three people with haemophilia A develop inhibitors to standard factor VIII replacement therapies, which limits treatment options and increases the risk of life-threatening bleeds and repeated bleeds, particularly in joints, that cause long-term damage. The disorder is due to patients not having enough factor VIII that combines factors IXa and X in the clotting cascade.

The Biologics License Application for emicizumab prophylaxis is based on findings from the phase 3 HAVEN 1 study and interim results from the Phase 3 HAVEN 2 study, according to a press release.

A Prescription Drug User Fee Act (PDUFA) action date of February 23, 2018 has been set by the FDA to make a decision on approval.

Emicizumab, a once weekly, ready-to-use solution for SC injection, is an investigational bispecific monoclonal antibody created to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process.

Data from the HAVEN 1 and 2 studies have also been submitted to the European Medicines Agency (EMA), per Roche, to be reviewed and assessed. Patients previously treated with on-demand BPAs were randomized 2:1 to receive either emicizumab prophylaxis (Arm A) or no prophylaxis (Arm B). The median age was 28 years. The primary endpoint was the number of treated bleeds over time with emicizumab prophylaxis compared with no prophylaxis, according to the release. Only 6% of patients in the control group experienced 0 bleeding events.

Throughout the study, 198 adverse events (AE) were reported in 103 participants receiving emicizumab prophylaxis, the most frequently-reported of which were injection-site reactions, which occurred 15% (28 occurrences in 15 patients) of the time.

It is being tested in pivotal phase 3 studies in people 12 years of age and older, both with and without inhibitors to factor VIII, and in children younger than 12 years of age with factor VIII inhibitors.

In a news release, Sandra Horning, M.D., chief medical officer and head of Global Product Development at Genentech said, "Results of our Phase III study in adults and adolescents as well as early Phase III results in children showed that emicizumab has significant potential to help people with hemophilia A with inhibitors, who face major challenges in preventing and treating bleeds". "We are working with the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible".

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