The US Food and Drug Administration (FDA) has approved antihemophilic factor [recombinant] PEGylated-aucl (Jivi, Bayer) for the routine prophylactic treatment of hemophilia A in previously treated adults and in children and adolescents aged 12 years or older.
Jivi is also indicated for on-demand treatment and the perioperative management of bleeding in the same patient population.
Jivi replaces the reduced or missing factor VIII in patients with hemophilia A. Through its site-specific PEGylation, Jivi has a half-life of 17.9 hours that delivers sustained levels in the blood, the company explained in a news release.
The initial recommended prophylactic regimen for Jivi is twice weekly; it can be administered every 5 days and can be further adjusted individually to less or more frequent dosing, based on bleeding episodes.
"As a physician who treats hemophilia A patients with a range of individualized needs, Jivi's approved dosing allows me to adjust frequency based on their bleed episodes to maintain protection from bleeds, which is a serious concern among patients," said Mark Reding, MD, lead investigator of the PROTECT VIII phase 2/3 study, which was the basis of FDA approval.
"Jivi is a welcome option that addresses a growing patient need to integrate treatment with personal lifestyles," said Reding, of the University of Minnesota, Minneapolis.
The PROTECT VIII trial included prophylactic dosing, on-demand treatment, and perioperative management of adults and of children and adolescents aged 12 years or older with severe hemophilia A. The trial demonstrated the drug's ability to protect against bleeding episodes for up to a median of 1.9 years, the company said.
Jivi was well tolerated in the majority of adult and adolescent patients in clinical trials. The most common adverse reactions in previously treated patients aged 12 years or older were headache, cough, nausea, and fever. A factor VIII inhibitor (1.7 BU/mL) was reported in one previously treated adult patient. Repeat testing did not confirm the presence of a factor VIII inhibitor, the company said.
Bayer has submitted marketing authorization applications for Jivi for the treatment of hemophilia A in the European Union and Japan.
Cite this: FDA Clears New Hemophilia A Drug With Flexible Dosing - Medscape - Aug 30, 2018.