Jivi (previously BAY 94-9027) is an approved prophylaxis or preventative treatment for hemophilia type A patients age 12 or older. Developed by Bayer, it is administered as an infusion into the bloodstream and can also be used on demand in response to a bleed.

How Jivi works

Hemophilia type A is an inherited condition, caused by a mutation in the coagulation factor 8, or F8 gene. This gene provides the instructions for a protein called factor 8 (F8) in the blood, which is essential for the correct initiation of blood clotting. If F8 is dysfunctional or not produced, this can result in excess bleeding both externally and internally and cause permanent damage or be fatal if not managed.

Jivi is a replacement therapy for the missing F8 protein in hemophilia type A and allows blood to clot normally. The therapy contains a recombinant F8 protein, or an F8 protein engineered to have an additional substance called polyethylene glycol (PEG) attached to it. This modification increases the stability of F8 and allows it to remain functional in the blood for longer compared to other F8 replacement therapies. As Jivi remains stable in the blood for an extended time, it requires fewer injections per week to help blood clot and prevent excessive bleeding in patients.

Jivi in clinical trials

Jivi has been tested in multiple clinical trials. An initial eight-week Phase 1 study (NCT01184820) that enrolled 14 hemophilia type A patients assessed the safety of Jivi, and how quickly the body breaks it down. The results of the study, published in the Journal of Thrombosis and Haemostasis, demonstrated that the treatment was well-tolerated and had an extended half-life of 19 hours on average. The half-life of a compound is the time it takes for half of it to be broken down by the body.

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 at sites in North and South America, Europe, and Asia. It aimed to assess Jivi’s safety and efficacy as both an on-demand and preventative treatment used at varying frequencies. Initially, all patients being treated preventatively received two infusions per week at a lower dose for 10 weeks. After that, they 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 were published in the Journal of Thrombosis and Haemostasis and showed that of the 702 bleeds that occurred during the trial, 636 (90.6 percent) were successfully controlled by two or fewer infusions of Jivi per week. No serious side effects were reported, and no patient developed inhibitors that would prevent Jivi from working.

A second Phase 3 multicenter, open-label, non-randomized trial (NCT01775618) called PROTECT-VIII Kids is assessing the safety and efficacy of Jivi in hemophilia type A patients younger than 12 years. The final results of the trial have not yet been published. However, initial results were presented at the 2016 World Congress of the World Federation of Hemophilia. A total of 32 patients, four younger than age 6 and 28 ages 6 to 12, were enrolled. Jivi controlled 92 percent of bleeds with two or fewer infusions per week. Eight patients discontinued treatment following a suspected immune response to Jivi, but no further safety concerns were raised.

Other information

The U.S. Food and Drug Administration (FDA) approved Jivi as a hemophilia type A prophylaxis treatment in August 2018.

The most common side effects of Jivi include headaches, coughing, nausea, and fever. More severe side effects, such as an allergic reaction to Jivi, are also possible.

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