Jivi antihemophilic factor (previously BAY 94-9027) is an approved prophylactic or preventive treatment for hemophilia type A patients, ages 12 or older. Developed by Bayer, it is given as an infusion into the bloodstream and also can be used on-demand in response to a bleed.
How does Jivi work?
Hemophilia type A is an inherited condition that results due to a mutation in the coagulation factor 8, or F8 gene. This gene provides the instructions for a protein called factor VIII (FVIII) in the blood, which helps blood to form clots. If FVIII is impaired or missing, this can result in excess bleeding both externally and internally. This can cause permanent damage or be fatal without treatment.
Jivi is a replacement therapy for the missing FVIII protein in hemophilia type A patients. It contains a recombinant FVIII protein, or an FVIII protein engineered to have an additional substance called polyethylene glycol (PEG) attached to it. This modification increases the stability of FVIII and allows it to remain functional in the blood longer compared with other FVIII replacement therapies.
Jivi in clinical trials
An initial Phase 1 study (NCT01184820), which enrolled 14 people with hemophilia type A, assessed Jivi’s safety and pharmacokinetics — a drug’s movement in the body. The results were published in the Journal of Thrombosis and Haemostasis and demonstrated that the participants tolerated the treatment well. The therapy’s average extended half-life was 19 hours. The half-life of a compound is the time it takes the body to break down half of it.
An open-label, randomized Phase 2/3 trial (NCT01580293) called PROTECT-VIII was key in Jivi’s approval as a treatment for hemophilia type A. It enrolled 134 patients with severe hemophilia type A. It aimed to assess Jivi’s safety and efficacy as both an on-demand and preventive treatment used at varying frequencies. Initially, all participants being treated preventively received two infusions per week at a lower dose for 10 weeks. They then received either two infusions per week at a low dose, one infusion every five days at a medium dose, or one infusion per week at a higher dose.
The results from that study, also published in the Journal of Thrombosis and Haemostasis, showed that two or fewer infusions of Jivi per week successfully controlled 636 (90.6%) of the 702 bleeds that occurred during the trial. The researchers reported no serious side effects, and no patient developed inhibitors that would prevent Jivi from working.
The PROTECT-VIII study also investigated Jivi’s effect on perioperative bleeding, or bleeding during and after surgery. The results appeared in the journal of Thrombosis Research and showed that in 26 major surgeries, Jivi was effective and well-tolerated by patients.
Long-term results of the PROTECT VIII open-label extension study appeared in the journal Haemophilia. They showed that 20.6% of patients in the prophylaxis group did not bleed at all during the extension period and 44.5% did not bleed for the six months prior to the interim result analysis in January 2018.
Ongoing clinical trials
A Phase 3 trial (NCT01775618) called PROTECT-VIII Kids is assessing the safety and efficacy of Jivi in boys younger than age 12 with hemophilia type A. The researchers have not yet published the final results of the multicenter, open-label, non-randomized trial. However, they presented initial results at the 2016 World Congress of the World Federation of Hemophilia.
In all, they enrolled 32 patients, including four boys younger than age 6, and 28 ages 6 to 12. Jivi controlled 92% of bleeds — 129 of 140 occurrences — with two or fewer infusions per week. Eight patients discontinued treatment following a suspected immune response to Jivi, but there were no further safety concerns.
Bayer now is recruiting an estimated 26 participants for a Phase 4 post-marketing study (NCT04085458) to gain more information about Jivi’s safety and effectiveness. Enrollment currently is open in Denmark, Bulgaria, Greece, Italy, Norway, and Poland, with additional sites possible in Spain.
Participants will start the study with a 45 international units (IU) per kg of body weight dose every five days. They then may continue on that dosage or change to twice-a-week treatment or therapy every seven days. Depending on their dosing frequency, patients will remain in the trial for one to two years. The researchers will monitor for any antibodies the body produces against Jivi. They also will record information about bleeding episodes and quality of life. The study will conclude in May 2022.
The U.S. Food and Drug Administration approved Jivi as a hemophilia type A prophylaxis treatment in August 2018.
Its most common side effect is fever, although headaches, coughing, and nausea have been reported. More severe side effects, such as an allergic reaction to Jivi, also are possible.
Last updated: Mar. 9, 2020
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