- Roche AG's highly anticipated hemophilia drug emicizumab has gained an early approval from the Food and Drug Administration as prophylaxis for bleeding episodes in patients who have hemophilia A with Factor VIII inhibitors.
- The drug, which Roche will market under the brand name Hemlibra, holds a once-weekly dosing schedule, putting it at the forefront of a wave of longer-acting hemophilia drugs that have entered the market in the last few years.
- But Hemlibra won't come cheap. Roche priced the product at a wholesale acquisition cost of $482,000 for the first year of treatment, after which it will cost slightly less depending on the weight of the patient, a company spokesperson said in an emailed statement.
Pharmaceutical executives and analysts have for some time looked at Hemlibra as a potential blockbuster and market disruptor. Hemophilia drugmakers Shire plc and Novo Nordisk A/S, for instance, expect their respective Feiba and NovoSeven franchises could be exposed once Roche's drug launches.
The thumbs up on Hemlibra comes three months ahead of the Feb. 23 target action date set for the drug's Biologics License Application. While most drugmakers welcome early approval, Roche may be particularly upbeat.
That's because the big pharma is facing biosimilar threats to its three best-selling drugs. Copycat versions of Rituxan (rituximab) are starting to eat away revenue of the reference product in Europe, where it's sold as MabThera. During the first nine months of 2017, MabThera sales totaled CHF 1.33 billion (about $1.34 billion) in Europe, down 6% from same period a year prior.
Cancer drugs Avastin (bevacizumab) and Herceptin (trastuzumab) are also seeing biosimilar competition begin to creep in. Combined, the trio of treatments accounted for roughly half of the CHF 10.1 billion (about $10.2 billion) that Roche's pharmaceutical division raked in during the third quarter.
Roche expects new portfolio additions will help offset some of the impending declines. Recently launched products like cancer med Tecentriq (atezolizumab) and multiple sclerosis treatment Ocrevus (ocrelizumab) are already fetching solid returns. Analysts predict Hemlibra will do the same; investment bank Jefferies, for example, pegged peak annual sales of $5 billion for the drug earlier this year.
That estimate, along with the FDA's approval, hinged on powerful data from the Phase 3 HAVEN 1 and HAVEN 2 studies. In the former, patients who took Hemlibra demonstrated a statistically significant 87% reduction in treated bleeds compared to those who didn't receive the therapy. In the later, which evaluated children, an interim analysis found that after a median 38 weeks, 87% of the participants on Roche's drug didn't experience any treated bleeds.
"People with haemophilia A who develop inhibitors face significant challenges preventing bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families, " Guy Young, director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles, said in a Nov. 16 statement from Roche. "This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week."
Perhaps even more important than its convenience factor, Hemlibra works on a once-weekly dosing schedule — a huge deal for hemophilia patients who, up until recently, were hampered with daily treatments. Drugmakers in the space have been advancing longer-acting therapies through the clinic and into the market over the past few years, but few have been able to offer drugs administered as infrequently as once per week.
Novo Nordisk predicts 50% of NovoSeven (coagulation Factor VIIa [recombinant]) sales may eventually become exposed to competition from Hemlibra, whereas Shire expects new hemophilia treatments — including, though not solely, emicizumab — to erode about half of its Feiba franchise by 2022.
Hemlibra's competitive advantage could be blunted, however, by a black box warning for thrombotic microangiopathy and thromboembolism included in its label. The warning puts Roche's drug on a more even playing field with NovoSeven and Feiba, which have their own black box warnings as well.