The FDA granted Sanofi’s hemophilia drug breakthrough status, as the French pharma continues pushing the therapy toward market as a potential rival to Roche’s blockbuster Hemlibra.
The regulator based the decision on data from the phase 3 XTEND-1 study, which showed the drug, called emonstrate efanesoctocog alfa, helped prevent bleeds in people with severe hemophilia A over a 52-week period.
Sanofi said it expects to share more data from the study at an upcoming medical meeting, and the phase 3 findings will serve as the basis for a mid-year FDA submission.
“The breakthrough therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with hemophilia A by providing higher protection for longer duration,” said John Reed, M.D., PhD, Sanofi’s global head of research and development, in a Wednesday release. “We are excited to work with regulatory authorities during the filing and review of this innovative therapy.”
If approved, efanesoctocog alfa will give the company a chance to catch up with Roche, having seen the Swiss pharma’s Hemlibra eat into sales of Sanofi’s current long-acting factor VIII therapy.
Efanesoctocog alfa, which Sanofi is developing with its parter Sobi, is designed to address the limitations of current hemophilia treatments. Existing factor VIII treatments require patients to undergo multiple intravenous infusions a week. Those infusions provide high protection, enabling patients to be more active, but still leave them at risk when factor VIII levels are at their trough level or if they miss an infusion. In contrast, Roche’s Hemlibra is given less frequently, but many patients still suffer acute bleeds that require additional factor therapy.
Sanofi, which acquired efanesoctocog alfa in its buyout of Bioverativ, will share commercialization with Sobi, which has rights in Europe, North Africa, Russia and most Middle Eastern markets.