Photo courtesy of Sobi
Efmoroctocog alfa (Elocta) packaging
The Saudi Food & Drug Authority (SFDA) in the Kingdom of Saudi Arabia has approved efmoroctocog alfa (Elocta®), a recombinant human factor VIII Fc-fusion protein, for the treatment of hemophilia A.
Efmoroctocog alfa is indicated for both on-demand treatment and prophylaxis in hemophilia A patients of all ages.
It is the first extended half-life and recombinant factor VIII Fc fusion protein therapy approved for the treatment of hemophilia A in Saudi Arabia.
The SFDA’s approval of efmoroctocog alfa was based on data from a pair of phase 3 studies: A-LONG and Kids A-LONG.
A-LONG
The A-LONG study included 165 previously treated males 12 years of age and older with severe hemophilia A. Researchers evaluated individualized and weekly prophylaxis to reduce or prevent bleeding episodes and on-demand dosing to treat bleeding episodes.
Prophylaxis with efmoroctocog alfa resulted in low annualized bleeding rates, and a majority of bleeding episodes were controlled with a single injection of efmoroctocog alfa.
None of the patients developed neutralizing antibodies, efmoroctocog alfa was considered well-tolerated, and the product had a prolonged half-life when compared with recombinant factor VIII.
Kids A-LONG
The Kids A-LONG study included 71 boys (younger than 12) with severe hemophilia A who had at least 50 prior exposure days to factor VIII therapies.
The children saw their median annualized bleeding rate decrease with efmoroctocog alfa, and close to half of the children did not have any bleeding episodes while they were receiving efmoroctocog alfa.
None of the patients developed inhibitors, and researchers said adverse events were typical of a pediatric hemophilia population.
Efmoroctocog alfa is being developed and commercialized by Sobi and Bioverativ.
