FDA approves Bayer's Kogenate FS antihemophilic factor VIII to prevent bleeding episodes in adults with hemophilia A

Bayer HealthCareMay 12 2014

Bayer HealthCare announced today that the U.S. Food and Drug Administration (FDA) has approved a new indication for Bayer's Kogenate® FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A. The approval is based on data from the SPINART study, in which 84 patients ages 15 to 50 were randomized to either prophylaxis (25 IU/kg three times per week) or on-demand treatment.

"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, M.D., Principal Investigator of the study and Director, Mountain States Regional Hemophilia and Thrombosis Center, University of Colorado at Denver and Health Sciences Center. "Such clinical information can help healthcare professionals provide appropriate treatment advice to their patients."

Data from the SPINART Study
The approval is based on data from the SPINART study¹, published in the Journal of Thrombosis and Haemostasis, in which 84 patients ages 15 to 50 were randomized to either prophylaxis (25 IU/kg three times per week) or on-demand treatment. In the study, patients were stratified based on target joints (presence/absence) and the number of bleeding events in the previous six months (≥15 vs. <15 annualized bleeds).  Safety and efficacy results from an analysis of the primary endpoint of bleeding frequency after a median follow-up period of 1.4 years provided the basis for the approval.   The safety objectives were also met.  Adverse events were consistent with the existing safety profile for Kogenate® FS.

As noted in the Prescribing Information, serious adverse reactions seen with Kogenate FS are systemic hypersensitivity reactions including bronchospastic reactions and/or hypotension and anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.  The most common adverse reactions (≥4%) observed in clinical trials were inhibitor formation in previously untreated and minimally treated patients, skin-related hypersensitivity reactions (e.g., rash, pruritus), infusion site reactions (e.g., inflammation, pain), and central venous access device (CVAD) associated infections.

Patients who received prophylaxis experienced significantly fewer bleeds (p<0.0001) compared to patients treated on-demand regardless of baseline subgroups examined including age, bleeding history, and presence or absence of target joints. The ratio of the mean bleeding frequency was 15.2 (95% CI: 8.5, 27.2; p<0.0001) for on-demand versus prophylaxis, indicating that patients who received on-demand treatment experienced on average 15.2 times as many bleeds compared to patients treated with prophylaxis.  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 (bleeds/subject/year) in the on-demand group was 33 versus 0 in the prophylaxis group.  Twenty-two of 42 (52%) prophylaxis subjects experienced no bleeding, and 12 of 42 (29%) prophylaxis subjects experienced only 1-2 bleeds during the follow-up period. 

Indications
Kogenate® FS antihemophilic factor (recombinant) is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A.

Kogenate FS is used to prevent or control bleeding in adults and children with hemophilia A. Your healthcare provider may give you Kogenate FS when you have surgery. Kogenate FS can reduce the number of bleeding episodes in adults and children when used regularly (prophylaxis). Kogenate FS can reduce the risk of joint damage in children without pre-existing joint damage when used regularly.

Kogenate FS is not used to treat von Willebrand disease.

Important Safety Information
You should not use Kogenate FS if you are allergic to rodents (like mice and hamsters) or are allergic to any ingredients in Kogenate FS.

Tell your healthcare provider if you have been told you have heart disease or are at risk for heart disease.

You could have an allergic reaction to Kogenate FS. Call your healthcare provider right away and stop treatment if you get rash or hives, itching, tightness of the chest or throat, difficulty breathing, light-headed, dizziness, nausea or a decrease in blood pressure.

Your body can make antibodies, called "inhibitors," against Kogenate FS, which may stop Kogenate FS from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to factor VIII.

Other common side effects of Kogenate FS are local injection site reactions (pain, swelling, irritation at infusion site) and infections from implanted injection device. Tell your healthcare provider about any side effect that bothers you or does not go away.

Call your healthcare provider right away if bleeding is not controlled after using Kogenate FS.