The US Food and Drug Administration (FDA) has approved a new indication for the recombinant antihemophilic factor VIII product octocog alfa (Kogenate).
It is now approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A.
Octocog alfa was already FDA-approved for the control and prevention of bleeding episodes in adults and children with hemophilia A, for perioperative management in adults and children with hemophilia A, as routine prophylaxis in children with hemophilia A, and to reduce the risk of joint damage in children without pre-existing joint damage.
The FDA’s latest approval is based on data from the SPINART study, in which patients were randomized to receive prophylactic octocog alfa or on-demand treatment. The study was sponsored by Bayer Healthcare, the company developing octocog alfa.
“In Bayer’s SPINART study, adult patients with hemophilia A on the prophylactic regimen experienced significantly fewer bleeding events than those using on-demand treatment,” said Marilyn Manco-Johnson, MD, principal investigator of the study and director at of the Mountain States Regional Hemophilia and Thrombosis Center at the University of Colorado at Denver and Health Sciences Center.
Results of the SPINART study were presented at the World Federation of Hemophilia 2012 World Congress and published in The Journal of Thrombosis and Haemostasis.
The study included 84 patients, ages 15 to 50, with hemophilia A. They were randomized to a prophylaxis regimen of 25 IU/kg 3 times per week (n=42) or on-demand treatment (n=42). Escalation by 5 IU/kg (to 30 or 35 IU/kg maximum) was allowed for subjects with 12 or more annual bleeds after 1 and 2 years.
Patients were stratified based on target joints (presence/absence) and the number of bleeding events in the previous 6 months (≥15 vs <15 annualized bleeds).
Patients who received prophylaxis experienced significantly fewer bleeds (P<0.0001) than patients treated on demand, regardless of factors such as age, bleeding history, and the presence or absence of target joints.
The ratio of the mean bleeding frequency was 15.2 (P<0.0001) for on-demand vs prophylaxis, indicating that patients who received on-demand treatment experienced, on average, 15.2 times as many bleeds as patients treated prophylactically.
The mean annualized bleed rates (bleeds/subject/year) were 37 in the on-demand group versus 2 in the prophylaxis group. The median annualized bleed rate in the on-demand group was 33 versus 0 in the prophylaxis group.
Fifty-two percent (22/42) of prophylaxis subjects experienced no bleeding, and 29% (12/42) of prophylaxis subjects experienced 1 to 2 bleeds during the follow-up period.
Adverse events were consistent with the existing safety profile for octocog alfa. The most common adverse reactions (≥4%) were inhibitor formation in previously untreated and minimally treated patients, skin-related hypersensitivity reactions (eg, rash, pruritus), infusion-site reactions (eg, inflammation, pain), and infections associated with a central venous access device.
Serious adverse reactions associated with octocog alfa include systemic hypersensitivity reactions—bronchospastic reactions and/or hypotension and anaphylaxis—and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
For more details on octocog alfa, see the full prescribing information.
