Bayer HealthCare has received approval from the US Food and Drug Administration (FDA) for a new indication of its Kogenate FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with haemophilia A.
FDA approval is based on a SPINART study, which 84 patients aged 15 to 50 were randomised to either prophylaxis (25 IU/kg three times per week) or on-demand treatment.
University of Colorado Mountain States Regional Hemophilia and Thrombosis Center director and principal investigator of the study Marilyn Manco-Johnson said in Bayer’s SPINART study, adult patients with haemophilia A on the prophylactic regimen experienced significantly fewer bleeding events than those using on-demand treatment.
"Such clinical information can help healthcare professionals provide appropriate treatment advice to their patients," Manco-Johnson said.
In the SPINART study, patients were stratified based on target joints (presence/absence) and the number of bleeding events in the previous six months (=15 vs. <15 annualised bleeds).
The company said that 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 trial met its primary efficacy and safety objectives for the treatment of adult patients with haemophilia A, while adverse events were consistent with the existing safety profile for Kogenate FS.
Most common adverse reactions (=4%) observed 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 (CVAD) associated infections.
SPINART (‘Secondary Prophylaxis in Adults, a Randomized Trial’) is an open-label, randomised, parallel-group study, in which subjects were randomized 1:1 to receive Kogenate FS either as a prophylaxis regimen of 25 IU/kg 3 times per week or on-demand.
Haemophilia A is a largely inherited bleeding disorder, which involves one of the proteins needed to form blood clots in the body being missing or reduced.
It is the most common type of haemophilia and is characterised by prolonged or spontaneous bleeding, especially into the joints, muscles or internal organs.
Image: Deficiency in coagulation factor VIII is the cause of haemophilia A. Photo: courtesy of ProteinBoxBot.