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FDA grants priority review to emicizumab for hemophilia A
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The FDA granted priority review to emicizumab for the treatment of adults, adolescents and children with hemophilia A with factor VIII inhibitors, according to the drug’s manufacturer.
Emicizumab (ACE910; Genentech, Roche) is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X.
“Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” Sandra Horning, MD, chief medical officer and head of global product development at Genentech, said in a company-issued press release. “Results of our phase 3 study in adults and adolescents — as well as early phase 3 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.”
The biologics license application for emicizumab included data from the randomized phase 3 HAVEN 1 study, which included adults and adolescents aged 12 years and older, as well as interim results from the phase 3 HAVEN 2 study, which included children aged younger than 12 years old.
The HAVEN 1 trial evaluated the efficacy, safety and pharmacokinetics of emicizumab prophylaxis compared with on-demand bypassing agents in 109 patients with hemophilia A with inhibitors to factor VIII. All patients had previously been treated with bypassing agents on demand or as prophylaxis.
Researchers randomly assigned patients 2:1 to emicizumab prophylaxis or no prophylaxis. Patients previously treated with prophylactic bypassing agents received emicizumab prophylaxis.
The number of treated bleeds over time served as the primary endpoint. Secondary endpoints included all bleed rate, joint bleed rate, spontaneous bleed rate, target joint bleed rate, health-related quality of life/health status, and intrapatient comparison to bleed rate on prior prophylaxis regimen with bypassing agents. Researchers also evaluated safety and pharmacokinetics.
Patients treated with emicizumab prophylaxis demonstrated an 87% reduction in treated bleeds (risk rate = 0.13; P < .0001).
The intrapatient analysis that compared the two prophylaxis regimens showed a 79% reduction in treated bleeds among a subset of patients treated with emicizumab.
Adverse events with emicizumab treatment included local injection site reactions, headache, fatigue, upper respiratory tract infection and joint pain.
The interim analysis of the single-arm, open-label HAVEN 2 trial — performed after a median 12 weeks of treatment — evaluated efficacy, safety and pharmacokinetics of once-weekly subcutaneous administration of emicizumab in 19 children with hemophilia A with inhibitors to factor VIII, who require treatment with bypassing agents.
Study objectives included the number of treated bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life and proxy health-related quality of life with aspects of caregiver burden.
Interim results appeared consistent with the positive results from the HAVEN 1 trial, according to the Genentech press release.
One of 19 children reported a treated bleed, and no joint or muscle bleeds were reported.
The most common adverse events included mild injection site reactions and common cold symptoms.
Results from both studies were presented in July at the International Society on Thrombosis and Haemostasis Congress.
The FDA set a target action date on the emicizumab application of Feb, 23, 2018.
“We are working with the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible,” Horning said in the release.
Additional studies designed to evaluate emicizumab in patients with hemophilia A — both with and without inhibitors — and to investigate less frequent dosing regimens are ongoing.