- Bayer on Thursday announced the U.S. approval of Jivi, a therapy for preventative and on-demand treatment of hemophilia A patients 12 years and older.
- Supporting the approval were data from the Phase 2/3 PROTECT VIII study, which administered prophylaxis Jivi either twice per week, once every five days or once every seven days, and on-demand Jivi when patients experienced bleeds. Results showed the median annualized bleed rate was less than one for 74% of patients who stayed on the once-every-seven-days regimen until the study's end.
- The Food and Drug Administration recommends patients taking Jivi to start with a twice-weekly regimen that can later be modified to a less frequent dosing schedule, such as once every five days. Bayer has two other marketed hemophilia A therapies: Kogenate, administered three to four times a week, and Kovaltry, administered two to four times a week, depending on the patient.
Historically, hemophilia treatments were administered every day or every other day. It's proven quite burdensome for patients, in turn putting pressure on drugmakers to create longer-acting and safer therapies, or risk losing market share.
Roche's newcomer Hemlibra (emicizumab) is already making waves on that front. It's approved for routine prophylaxis of hemophilia A patients with Factor VIII inhibitors and comes as a once-weekly dose. Analysts expect the drug to take a big bite out of rival inhibitor-focused treatments from Shire and Novo Nordisk.
The market shake-up is likely to get bigger too. Recent Phase 3 data puts Hemlibra in a good position to get an FDA OK later this year for hemophilia A patients without inhibitors. Further down the line, gene therapies could offer a one-time treatment for the uncommon blood disorder. BioMarin Pharmaceutical, Spark Therapeutics and Bayer are a few of the companies working on such therapies.
Competition, both current and future, will further test Bayer's foothold in hemophilia, which recently became shakier due to a terminated distribution agreement at the end of last year. The German drugmaker reported revenue from its antihemophilic factor therapies Kogenate and Kovaltry of 967 million euros in 2017, down 17% year over year due to lower order volumes from that distributor.
Bayer's new product may therefore provide a welcome near-term boost to its hemophilia business. In addition to a benefit on annualized bleed rates, the PROTECT VIII study found more than 90% of bleeds were controlled with two or less infusions of Jivi (antihemophilic factor [recombinant] PEGylated-aucl).
The therapy's flexible dosing is also an upside, according to Bayer.
"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," Carsten Brunn, president of Bayer Pharmaceuticals' Americas Region, said in an Aug. 30 statement.