FDA Approves Hemophilia A Treatment, BAY94-9027

This morning, August 30, 2018, the US Food and Drug Administration (FDA) approved Bayer’s antihemophilic factor [recombinant] PEGylated-aucl, BAY94-9027 (Jivi), for routine prophylactic treatment of hemophilia A in previously treated adults and adolescents aged 12 years or older.

“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, PROTECT VIII lead investigator and associate professor of medicine at the University of Minnesota, in a recent statement. “Jivi [BAY94-9027] is a welcome option that addresses a growing patient need to integrate treatment with personal lifestyles.”

For BAY94-9027, the initial recommended prophylactic regimen is twice-weekly (30-40 IU/kg) with the ability to dose every 5 days (45-60 IU/kg). Based on bleeding episodes in the individual, dosing can be further adjusted.

Data from the pivotal, 36-week, international, open-label, phase 2/3 PROTECT VIII trial conducted in previously treated adults and adolescents 12 years of age or older with severe hemophilia A served as the basis for the approval. Among the hallmarks, BAY94-9027 showed bleed protection and safety of up to a median of 1.9 years (range of 0 to 2.6 years).

Pharmacokinetics, efficacy, and safety of BAY94-9027 for on-demand treatment of bleeds and for prophylactic therapy at different dosing regimens were assessed in Part A of the trial. For individuals who completed Part A, an optional extension study was made available for a minimum 100 accumulated exposure days. Safety and efficacy of BAY94-9027 during major surgery was assessed in Part B.

A headache, cough, nausea, and fever were the most commonly experienced adverse events. Overall, however, treatment with BAY94-9027 was found to be well-tolerated the majority of adult and adolescent patients. In previously treated adult participant, a FVIII inhibitor (1.7 BU/mL) was reported; however, its presence was not confirmed upon repeat testing.

"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," added Carsten Brunn, president of Bayer Pharmaceuticals, Americas Region. "Jivi's [BAY94-9027] 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 [BAY94-9027] in other regions around the world.”

BAY94-9027 is now the third approved treatment for patients with hemophilia A.