- The EU has given a thumbs up to Roche AG's Hemlibra as a prophylaxis for bleeding episodes in hemophilia A patients with Factor VIII inhibitors.
- Data from the HAVEN 1 and HAVEN 2 studies supported Hemlibra's approval both in Europe and the U.S. The former study, which enrolled hemophilia A patients 12 years and older with Factor VIII inhibitors, found those taking Roche's drug as a preventative treatment showed an 87% reduction in treated bleeds. The latter, which enrolled children under 12 with the same condition, found 87% of participants taking the drug experienced no treated bleeds.
- The approval decision came a little more than a month after the European Medicine Agency's Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion about the drug. While the Commission isn't required to follow the committee's recommendation, it nearly always does.
Analysts expect Hemlibra (emicizumab) to be one monster of a revenue driver. Investment bank Jefferies, for instance, estimated last year that the drug would hit peak annual sales of as much as $5 billion.
But such a feat would require the drug's label to expand beyond hemophilia A patients with inhibitors to treat a much wider population.
Roche is rapidly advancing on that front.
By the end of 2018, the Swiss pharma plans to file Hemlibra for U.S. and European approval as a treatment for hemophilia A patients without inhibitors — supported by positive results from HAVEN 3, a late-stage trial testing the drug on a one- to two-week dosing regimen. Given that roughly 70% of people with severe hemophilia A don't have inhibitors, such approvals would open up Hemlibra to a much larger pool of potential patients.
Also this year, the company is looking to get Europe and FDA to green light a four-week dosing schedule for Hemlibra in patients with and without inhibitors.
"In terms of the filing, as you can imagine, we’re actively putting the files together until now, which will include both HAVEN 3, which is the non-inhibitor data, and also HAVEN 4, which will allow us to look at different dosing intervals. We intend to file as soon as we can this year," Roche CEO Daniel O'Day said during the company's fourth quarter earnings call earlier this month.
In the meantime, Hemlibra's impending European launch will likely offset Roche's recent struggles there.
The drugmaker's pharmaceutical sales in Europe decreased 2% year over year in 2017, mostly because biosimilar competition began eating away at the MabThera/Rituxan (rituximab) franchise, which fell 11%.