Roche's Hemlibra, eyeing a big new market, puts up standard-busting bleed data

Here comes Hemlibra. The Roche drug, on its way to treating a much wider population than the one it’s now approved for, put up some big data over the weekend that should back its claim.

In a phase 3 study in hemophilia A patients without factor VIII inhibitors, Hemlibra pared down bleeding incidents that needed treatment by 96% compared with no preventive therapy, Roche said Monday. The results covered doses administered every week or two weeks.

In a subset of patients who had previously received factor VIII prophylaxis, which is the standard of care, Hemlibra cut bleeds by 68%. The results made it the first drug to top factor VIII at reducing bleeding episodes.

Those are big numbers for Hemlibra, which is awaiting word from the FDA on its bid for approval in hemophilia patients without factor VIII inhibitors. Those inhibitors develop in some patients during factor VIII therapy. Right now, Hemlibra bears a nod only in patients who do have inhibitors, a much smaller group, but last month it picked up a “breakthrough” tag to speed its way toward approval in the larger population.

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If Hemlibra can land the new green light, it could generate $2 billion by 2025, analysts say, although some have predicted that tally could go even higher. Jefferies analysts, for instance, have set their 2025 sales prediction at $5 billion.

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Investors are expecting big things from Hemlibra, too—so much so that shares of competitor Shire have been taking a beating on Hemlibra’s success. Soon, that problem will be Takeda’s; the Japanese pharma agreed earlier this month to pony up $62 billion for the Irish drugmaker, which acquired its hemophilia portfolio with a big buy of its own, Baxalta.

Another reason Hemlibra’s prospects are so bright, aside from its efficacy? Convenience. A separate phase 3 trial, also presented Monday, showed that more than half—56.1%—of hemophilia A patients receiving Hemlibra just once every four weeks suffered no treated bleeds whatsoever, with 90.2% of patients experiencing three or fewer.

“These results demonstrate that Hemlibra administration every four weeks can provide clinically meaningful control of bleeding in people with hemophilia A,” whether or not they have factor VIII inhibitors, Roche said.