FDA Approves Bayer’s Jivi for Hemophilia A Patients 12 and Older

The U.S. Food and Drug Administration has approved Bayer’s Jivi, previously known as BAY94-9027, as a preventive therapy for hemophilia A in patients ages 12 and older, the company announced.

The recommended regimen is for Jivi to be administered intravenously (into the blood) two times a week (30-40 IU/kg), which can be extended to every five days (45-60 IU/kg). Individual adjustments can also be made depending on the frequency of bleeding episodes.

The FDA has also approved Jivi for on-demand treatment and the management of perioperative bleeding in patients ages 12 and older.

Bayer submitted its application seeking approval of Jivi in November 2017, supported by the positive results of the Phase 2/3 PROTECT VIII trial, which showed that Jivi protected from bleeds up to a median of 1.9 years — ranging from zero to 2.6 years.

“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,” Mark Reding, MD, lead investigator of the trial and associate professor of medicine at the University of Minnesota, said in a press release. “Jivi is a welcome option that addresses a growing patient need to integrate treatment with personal lifestyles.”

The PROTECT VIII study (NCT01580293) evaluated the effectiveness and safety of Jivi to treat bleeds, either on demand or for prevention.

Researchers tested several treatment frequencies depending on observed bleeding: once every five days, once every seven days, or twice weekly in patients 12 and older with severe hemophilia A.

A total of 141 patients were recruited. Those in the preventive treatment groups received the therapy for 36 weeks. Additionally, patients could extend treatment for at least 100 total exposure days, defined as the days on which infusions take place. Overall, 126 patients have completed the study.

Data first reported in 2014 showed that BAY94-9027 could achieve bleed protection with extended dosing intervals, and effectively resolved 91% of acute and breakthrough bleed events with one or two infusions.

Treatment with Jivi was generally safe and well-tolerated, with the most frequently reported adverse effects being headache, cough, nausea, and fever.

“Today’s approval builds on our 25-year partnership with the hemophilia community and underscores our commitment to developing new therapies that help meet the needs of patients living with this life-long disease,” said Carsten Brunn, president of Bayer Pharmaceuticals, Americas. “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.”

Jivi is a long-acting, lab-produced form of coagulation Factor VIII, a protein essential for blood clotting, which is missing or deficient in hemophilia A patients.

The therapy was designed to improve the lifetime of factor VIII — current available factor VIII therapies have low stability, making factor VIII administration necessary every other day or three times per week.

To overcome this limitation, researchers added a small compound called PEG, or polyethylenglycol, to the factor VIII protein in Jivi. This modification extends the stability of the protein circulating in the blood while preserving its coagulation activity.

Bayer has also filed for the treatment to be approved for use in patients in the European Union and Japan.