Positive Phase III results for Hemlibra for hemophilia A without factor VIII inhibitors

Positive pivotal data from the Phase III HAVEN 3 study, which evaluated Hemlibra (emicizumab) prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors, were published in the New England Journal of Medicine (NEJM) today, said the drug’s developer, Swiss pharma giant Roche (ROG: SIX).

"In the HAVEN 3 study, Hemlibra showed a significant and clinically-meaningful reduction in bleeds in people with hemophilia A without factor VIII inhibitors, while offering multiple subcutaneous dosing options," said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. "The publication of these results in the New England Journal of Medicine represents a major advance for hemophilia research and reinforces the potential of Hemlibra to change the standard of care for people with hemophilia A."

"Current prophylactic treatment options for people with hemophilia A can require frequent intravenous infusions, and despite treatment, many continue to have bleeds that can lead to long-term joint damage," said Sandra Horning, Roche's chief medical officer and head of global product development. "Given the challenges many people face managing their hemophilia, we believe Hemlibra could make a meaningful difference, and we are working with health authorities to hopefully make this treatment available to people with hemophilia A without factor VIII inhibitors as soon as possible."

Analysts at Jeffries have predicted that Roche’s Hemlibra could generate $5 billion in annual sales – provided its use for hemophilia A is widened to the larger, non-inhibitor population. In the first half of this year, Hemlibra generated sales of 57 million Swiss francs ($58.2 million).

Earlier this year, the US Food and Drug Administration granted Breakthrough Therapy designation and Priority Review to Hemlibra for people with haemophilia A without factor VIII inhibitors based on data from the HAVEN 3 study. The FDA is expected to make a decision on approval by October 4, 2018. In addition, the company's Marketing Authorization Application (MAA) variation of Hemlibra for hemophilia A without factor VIII inhibitors is under review by the European Medicines Agency (EMA). Submissions to other regulatory authorities around the world are ongoing.