FDA expands label for Roche's Hemlibra to include haemophilia A without factor VIII inhibitors

Roche said Thursday that it won FDA approval for Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A without factor VIII inhibitors. The therapy was first cleared in the US last November for haemophilia A patients who have developed resistance to factor VIII replacement therapy. 

Sandra Horning, chief medical officer at Roche, noted that "Hemlibra is now the only FDA-approved medicine for people with haemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies." 

Expanded approval was backed by data from the Phase III HAVEN 3 and HAVEN 4 studies. Results from HAVEN 3 showed that Hemlibra was linked to a significant and clinically meaningful reduction in the number of treated bleeds compared to no prophylaxis, while in HAVEN 4, the therapy demonstrated a clinically meaningful control of bleeding, Roche said.

Analysts have forecast sales of $2.6 billion for Hemlibra by 2022, with some estimating annual sales could eventually hit as much as $5 billion. According to a spokeswoman at Roche's Genentech unit, the annual list price for Hemlibra will be about $492 000 per year for a haemophilia A patient of average weight, following an initial first-year dose that costs an additional $38 000.

When the treatment was first approved last November, Roche had set its wholesale acquisition price at $482 000 for the first year of treatment and then about $448 000 the following year. Recently, while pledging not to implement further price hikes on its medicines for the remainder of this year, Roche noted that Hemlibra is priced at less than half the cost of the standard treatment for haemophilia A with inhibitors. 

Hemlibra was previously granted priority review and breakthrough therapy status for use in patients with haemophilia A without factor VIII inhibitors. 

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