FDA Grants Breakthrough Therapy Designation to Roche’s ACE910

The FDA has granted breakthrough therapy designation to Roche’s investigational ACE910 for the prophylactic treatment of patients 12 years and older with hemophilia A with factor VIII inhibitors.

Roche says it is preparing to initiate a Phase 3 trial in patients with this specific form of hemophilia by the end of this year, and a separate Phase 3 trial in patients without factor VIII inhibitors in 2016. A trial in pediatric patients with hemophilia A is also planned for next year.

Roche’s product poses potential competition to Baxalta’s flagship hemophilia product Advate (antihemophilic factor (recombinant)), says Morningstar. ACE910 could reach the market for inhibitor patients as early as 2018, beating Baxalta’s gene therapy treatments to market.

The hemophilia market is expected to grow to $11 billion next year, according to Morningstar. Hemophilia A represents the vast majority of the hemophilia market, worth about six to seven times the size of hemophilia B, S&P Capital IQ analyst Jeffrey Loo said. — Jonathon Shacat