The US Food and Drug Administration (FDA) has approved the recombinant factor VIII Fc fusion protein efmoroctocog alfa (Eloctate) to treat children and adults with hemophilia A.
Efmoroctocog alfa is intended to reduce the frequency of injections required to prevent or reduce the occurrence of bleeding in patients with hemophilia A.
The FDA approved efmoroctocog alfa based on results of the phase 3 A-LONG trial, which were reported last year.
The trial included 165 male patients age 12 or older.
Patients who received individualized prophylaxis with efmoroctocog alfa every 3 to 5 days reduced their annualized bleeding rate by 92% compared with patients who received episodic treatment.
Patients who received weekly prophylaxis with efmoroctocog alfa reduced their annualized bleeding rate by 76% compared to patients who received episodic treatment.
Overall, 1 injection of efmoroctocog alfa resolved 87.3% of bleeding episodes.
None of the patients in the study developed neutralizing antibodies, and there were no serious adverse events related to efmoroctocog alfa.
The study was published online in Blood last November.
Efmoroctocog alfa consists of the coagulation factor VIII molecule linked to an Fc protein fragment found in antibodies, which confers the product with a longer half-life than recombinant factor VIII.
Efmoroctocog alfa is specifically indicated for the control and prevention of bleeding episodes, the management of bleeding during surgical procedures, and prophylaxis against bleeding episodes.
The approval of efmoroctocog alfa fills an unmet medical need, according to Johnny Mahlangu, MD, of the Haemophilia Comprehensive Care Centre at the University of Witwatersrand in Johannesburg, South Africa.
He explained that the fusion protein allows patients with hemophilia A to go for longer intervals between prophylactic infusions while maintaining good control of bleeding episodes.
The product, which received orphan drug designation by the FDA, is manufactured by Biogen Idec, Inc, of Cambridge, Massachusetts.
Full prescribing information is available on the drug website.
