Roche’s fresh hemophilia drug Hemlibra dramatically reduced bleeding in a broad population of hemophilia patients, results by two clinical trials showed on Monday, setting of which up to take a dominant market position.
Hemlibra cut by 96 percent the incidence of treated bleeds in hemophilia A patients who did not get preventive treatment in addition to compared with patients who did get preventive treatment from the form of clotting factors of which reduced them by 68 percent.
The positive results by the two trials known as HAVEN 3 in addition to 4 included so-called non-inhibitor patients. Hemlibra’s initial success was in patients with inhibitors, which are antibodies of which cause resistance to replacement clotting factors.
Hemlibra’s current regulatory approval can be only for these inhibitor patients although Roche plans to submit the latest findings to authorities around the globe to widen its use.
While the drug’s latest success had been expected, the positive results seen in a wide range of patients should underpin demand for a medicine of which Roche can be relying on as several of its blockbuster cancer drugs face cut-cost rivals.
Jefferies analysts said HAVEN 4 also showed there was a potential to treat both inhibitor in addition to non-inhibitor patients on only a once-monthly basis. Currently, Hemlibra can be given as a once-weekly injection.
“of which sets Hemlibra up to become the fresh standard of care for hemophilia A, which we view as a $5 billion peak sales opportunity,” they said.
The current standard of care for people with hemophilia A without inhibitors can be replacement factor VIII clotting factor.
Hemlibra will shake up the market in addition to pose a threat to established players reliant on factor replacement therapies, notably Shire, which has agreed to be acquired by Takeda Pharmaceutical.
Also being challenged are Bayer, CSL, in addition to Novo Nordisk, as well as Sanofi, which earlier of which year bought U.S. hemophilia specialist Bioverativ.
fresh science also promises to bring further alterations from the years ahead, with several companies working on hemophilia gene therapy, in which a harmless virus can be used to introduce DNA to fix the faulty genes behind the disease, offering a possible one-off cure.
Data by the two Roche clinical studies were presented at the globe Federation of Hemophilia congress in Glasgow, Scotland.
“With of which data, we at of which point have positive results by all four of our Phase III trials of which reinforce the overall efficacy in addition to safety of Hemlibra in addition to its potential to improve care for all people with hemophilia A,” said Roche Chief Medical Officer Sandra Horning.