CHMP Backs First-in-Class Drug Emicizumab for Hemophilia A

CHMP Backs First-in-Class Drug Emicizumab for Hemophilia A

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization after accelerated assessment for emicizumab (Hemlibra, Roche Registration Limited), a first-in-class medication for the indication of preventing bleeding or reducing the frequency of bleeding episodes in patients of all ages with hemophilia A with factor VIII inhibitors, according to an EMA news release.

The usual treatment for hemophilia A is to replace the missing factor VIII; however, some patients develop inhibitors (antibodies) after taking these medications. These inhibitors decrease the effectiveness of medications and can make disease management much more difficult.

Emicizumab is the first monoclonal antibody for the treatment of patients with hemophilia A and inhibitors. The development of this medication represents a significant advance in hemophilia treatment, as there have been no new medications for the disorder in 20 years, according to the EMA release

“Many people with haemophilia A develop inhibitors, which greatly impacts their ability to treat or prevent bleeds and diminishes their quality of life,” said Brian O’Mahony, chief executive of the Irish Haemophilia Society and president of the European Haemophilia Consortium, in a Roche news release. “It’s been over 20 years since we last saw a new medicine for people with haemophilia A with inhibitors, so there is a pressing need for innovative treatments that control bleeding and decrease the negative impact on quality of life.”

Hemlibra is administered once weekly by subcutaneous injection, unlike current treatments, which must be given by medicines that bypass factor VIII and require frequent and prolonged infusions, according to EMA.

“Hemlibra has been shown to effectively reduce the frequency of bleeds compared to currently available medicines, and with once-weekly administration by injection under the skin, it could also greatly reduce the treatment administration burden, particularly for young children with haemophilia A with inhibitors and their families,” said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development, in the company release.

Safety, Efficacy

The EMA panel’s recommendation follows discussion of data from two phase 3 clinical trials: a randomized, open-label trial that included 109 patients aged 12 years of older, and an ongoing single-arm, open-label study that included results for 60 children younger than 12 years.

Overall, when used preventively in patients with hemophilia with inhibitors, emicizumab decreased bleeding episodes requiring treatment with coagulation factors by approximately 80% to 90% when compared with on-demand use of bypassing medications without prophylactic treatment.

The most frequently reported adverse events were injection-site reactions, headache, thrombotic microangiopathy, fever, diarrhea, and joint and muscle pain.

The CHMP’s decision is an intermediary step toward patient access. The European Commission will review the CHMP’s report and adopt a decision regarding an EU-wide marketing authorization. Once granted, price and reimbursement decisions will occur at the level of each member state after accounting for the potential role and use of emicizumab in the context of that country’s national health system.

The US Food and Drug Administration approved emicizumab on November 16, 2017.

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