FDA approves Kogenate FS for prophylaxis in hemophilia A

Bayer HealthCare announced the FDA expanded the approval of the company’s recombinant antihemophilic factor VIII to include routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A.

Bayer’s Kogenate FS received FDA approval based on data from the SPINART study, in which 84 patients aged 15 to 50 years were randomized to receive either prophylaxis (25 IU/kg three times per week) or on-demand treatment.

Marilyn Manco-Johnson

“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,” Marilyn Manco-Johnson, MD, principal investigator of the study and director of the Mountain States Regional Hemophilia and Thrombosis Center at the University of Colorado at Denver and Health Sciences Center, said in a press release. “Such clinical information can help health care professionals provide appropriate treatment advice to their patients.”

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 approval for this indication was based on safety and efficacy results from an examination of the primary endpoint of bleeding frequency after a median follow-up period of 1.4 years. The safety objectives were also met. Adverse events were consistent with the existing safety profile for Kogenate FS.

The most common adverse reactions (≥4%) associated with Kogenate FS in clinical trials were inhibitor formation in previously untreated and minimally-treated patients, skin-related hypersensitivity reactions, infusion site reactions, and central venous access device-associated infections.

Patients who received prophylaxis exhibited 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/patient/year) were 37 in the on-demand group vs. 2 in the prophylaxis group. The median annualized bleed rate (bleeds/patient/year) in the on-demand group was 33 vs. 0 in the prophylaxis group. In addition, 52% of prophylaxis patients experienced no bleeding, while 29% of prophylaxis patients experienced only 1-2 bleeds during the follow-up period.