In a phase 2/3 trial, the Bayer Pharmaceuticals therapy demonstrated protection from bleeding and patient safety for up to a median of 1.9 years.
The US Food and Drug Administration (FDA) has approved antihemophilic factor (recombinant) PEGylated-aucl (Jivi) for the prophylactic treatment of hemophilia A.
The therapy, from Bayer Pharmaceuticals, is indicated as a twice-weekly treatment for adults and adolescents aged 12 years or older with moderate-to-severe hemophilia A. It has also been approved as an on-demand therapy and for the perioperative management of bleeding in the same patient population.
With the approval, patients may be able to adjust dosing to once every 5 days, or for even more frequent dosing, based on bleeding episode rates. Jivi’s approval was based on the results of the phase 2 and 3 PROTECT VII trial, in which the therapy demonstrated protection from bleeding and patient safety for up to a median of 1.9 years.
PROTECT VIII was a 36-week, international, open-label study conducted in previously treated adults and adolescents 12 years of age or older with severe hemophilia A. An extension study was offered to patients who completed the initial phase of analysis, and assessed the therapy’s benefits over a minimum of 100 accumulated exposure days.
Approximately 1 in 5000 males are born with hemophilia, which currently affects about 400,000 people globally and 20,000 people in the US alone. The mostly inherited disease reduces or removes factor VIII, the proteins responsible for forming blood clots in the body. Current treatments have significantly improved patients’ life expectancies, however: in nearly a century, patients with hemophilia have improved to live 11.4 years longer than expected.
Jivi, the third therapy approved for hemophilia from Bayer, replaces the missing proteins in its treated patients. In its nearly 18-hour half-life, the therapy delivers sustained levels in the blood.
Mark Reding, MD, lead investigator of PROTECT VIII and an associate professor of medicine at the University of Minnesota, emphasized how the therapy’s approved dosing allows physicians such as himself to make adjustments based on patients’ bleeding episodes.
“Jivi is a welcome option that addresses a growing patient need to integrate treatment with personal lifestyles,” Reding said in a statement.
Bayer has also submitted applications for the drug’s marketing in the European Union and Japan. Carsten Brunn, president of Bayer, Americas Region, said Jivi’s FDA approval comes during the 25-year anniversary of the company’s work in the hemophilia community.
“Jivi’s proven efficacy with its unique dosing regimen is an important benefit to patients that we look forward to bringing to the global community, as we pursue additional regulatory approvals for Jivi in other regions around the world,” Brunn said.