The FDA approved Sanofi and Sweden-based Sobi’s efanesoctocog alfa, now marketed as Altuviiio, to treat bleeding in adults and children with hemophilia A.
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Thursday, the FDA approved Sanofi and Sweden-based Sobi’s efanesoctocog alfa, now marketed as Altuviiio, to treat routine prophylaxis and on-demand bleeding in adults and children with hemophilia A.
Altuviiio, a factor VIII replacement therapy, is also indicated for surgical management in these patients and is the first hemophilia A therapeutic to provide normal or near-normal levels of factor activity with once-weekly dosing.
Unlike other approved hemophilia A therapeutics on the market, Altuviiio is a recombinant Fc fusion protein that incorporates a region of the blood-clotting protein von Willebrand factor. This unique structure extends Altuviiio’s time in circulation and gives it a longer plasma half-life than other factor VIII products.
Altuviio’s approval is an important clinical advancement for hemophilia because it provides patients with an effective treatment option with a simplified weekly dose, Lynn Malec, M.D., associate professor of medicine and pediatrics, The Medical College of Wisconsin, said in a statement.
Data from the Phase III XTEND-1 study supported the FDA’s regulatory nod. Once-weekly prophylaxis with Altuviiio significantly lowered annualized bleeding rate (ABR) in patients with severe hemophilia A. When compared with prior factor prophylaxis, Altuviiio reduced ABR by 77%.
Moreover, XTEND-1 showed that Altuviiio could induce average factor VIII activity greater than 40% for most of the week, keeping its levels above 10% seven days after the dose.
This high and sustained increases in factor VIII activity levels after Altuviiio treatment have the potential to change the hemophilia landscape, Paul Hudson, CEO, Sanofi, said in a statement.
The FDA accepted Sanofi’s Biologics License Application (BLA) for efanesoctocog alfa in August 2022 and evaluated it under priority review, with an initial target action date of Feb. 28.
Efanesoctocog alfa had also previously been given the FDA’s Breakthrough Therapy and Fast Track designations.
Second in Hem A Portfolio
Altuviiio isn’t Sanofi and Sobi’s first hemophilia A treatment. The partners also own Eloctate, which Sobi developed with Biogen and won approval for in 2014.
In 2017, Biogen spun-out its hemophilia unit into a company dubbed Bioverativ, carrying both Eloctate and hemophilia B therapeutic Alprolix. Sanofi acquired Bioverativ in 2018 for $11.6 billion.
In 2017, Roche entered the hemophilia A space with an FDA approval of Hemlibra (emicizumab-kxwh), a bispecific antibody that serves as a proxy for factor VIII, bringing together factor IX and factor X to restore the coagulation cascade.
Also vying for a piece of the hemophilia A market is BioMarin, which resubmitted the BLA for its gene therapy valoctocogene roxaparvovec in October 2022 and has a target action date of Mar. 31.
