- In a decision that will substantially expand the market opportunity for Roche's Hemlibra, the Food and Drug Administration on Thursday approved the hemophilia A therapy for patients without inhibitors.
- Hemlibra first gained an FDA OK late last year for the treatment of hemophilia A patients with inhibitors — meaning they developed antibodies that attack infused clotting factor. Though more prominent in severe forms of the disease, these antibodies affect less than 10% of the mild-to-moderate hemophilia A population.
- Analysts expect Hemlibra, with its strong performance in the clinic and advantageous one-week dosing schedule, to shake up the hemophilia A market on its way to becoming a blockbuster. The drug has already proven a competitive threat to franchises like Novo Nordisk's NovoSeven and Shire's Feiba, and Thursday's approval in the non-inhibitor setting further opens the door for disruption.
Intensifying biosimilar competition to its top three cancer drugs has put pressure on Roche to revitalize its portfolio with new blockbusters. Hemlibra (emicizumab) could therefore provide some of the fresh blood the Swiss pharma needs.
"Even with these exposures ... we anticipate that Roche will still grow its earnings in the coming years due to growth in other products including Ocrevus for multiple sclerosis and Hemlibra for hemophilia," analysts from Moody's Investors Service wrote in an Oct. 4 note.
Supporting that conclusion, investment bank Jefferies has estimated sales of Hemlibra could eventually reach $5 billion a year — assuming it gained approval for the entirety of the hemophilia A population.
Analysts at Jefferies and elsewhere have defended those high estimates by pointing to both Hemlibra's efficacy and its once-weekly dosing regimen. In the Phase 3 HAVEN 3 study, for instance, hemophilia A patients 12 years and older without inhibitors who were put on once-weekly Hemlibra prophylaxis experienced a 96% reduction in treated bleeds compared to those who didn't receive preventative treatment. And for patients receiving Hemlibra every two weeks, there was a 97% reduction in treated bleeds versus no preventive treatment.
Notably, Hemlibra's updated label allows for once-every-two-week and once-every-four-week dosing, which is much less frequent than many of the hemophilia A therapies currently available, which are administered multiple times a week.
"Doing multiple times a week IV infusions is challenging. Adherence is not great, people are still bleeding," Michael Callaghan, a hematologist at Children's Hospital of Michigan who has also served as an advisor to Roche, told BioPharma Dive. "So to have a medicine that's much easier to use and with a package now that's even once a month dosing ... I think is just going to be a gamechanger."
As for physicians, Callaghan anticipates a mixed bag where some are quick to switch their patients over to Hemlibra while others take a more wait-and-see approach. In either case, he expects patients will drive much of the conversation.
"There's always a little bit of inertia. It's not like people beat down the doors to switch products in a day," he said. "But, I think this one has more advantages than maybe other changes we've seen in the past."
Hemlibra's pricing will remain the same through this new approval. For an average weight person with hemophilia A, the wholesale acquisition cost will be approximately $492,000 annually for each year after the first-year loading dose, which is about $38,000 more.
A Roche spokesperson said to BioPharma Dive in an interview that the company doesn't speculate on Hemlibra sales, but did note the drug had captured about 20% of the hemophilia A with inhibitors market as of the second quarter this year.
Others, meanwhile, have predicted that the drug could snag up to 50% of the hemophilia A without inhibitors market within three years of approval.
Hemlibra isn't without future competition though. Several companies, including BioMarin Pharmaceutical and Spark Therapeutics, are working on gene therapies to disrupt the hemophilia space even further.
Those therapies won't come for some time. And if they do, Roche says it encourages the competition.
"There really has not been any big innovation for a long, long time. The biggest innovation was sort of extending the half-life of the factors from 12 to 18 hours, so it's really exciting that there are a lot of companies working on things," Gallia Levy, associate group medical director at Roche's Genentech, told BioPharma Dive.
"It's still early for gene therapy and how it would fit in to they treatment landscape eventually. And how physicians would choose to use it — I think it's still to be determined," she added. "But anything that brings more therapies for patients, we welcome. I think it's going to be great if they have more choices."