FDA grants approval for Jivi in pediatric patients 7 to under 12 years of age with hemophilia A (congenital Factor VIII deficiency)- Bayer

 Bayer announced that the FDA  has approved Jivi, a recombinant DNA-derived, extended half-life factor VIII concentrate, for use in pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency). The approval is based on data from the Alfa-PROTECT and PROTECT Kids studies, demonstrating Jivi’s safety and efficacy in children aged 7 to under 12 years with severe hemophilia A. This significant milestone reflects Bayer’s ongoing commitment to providing effective treatment options for the hemophilia community.

Alfa-PROTECT Study was a multi-center, prospective, single-arm, study to evaluate the safety of Jivi infusions for prophylaxis and treatment of bleeding in previously treated pediatric patients 7 to <12 years of age with severe hemophilia A. The study evaluated the potential risk of hypersensitivity and loss of drug effect associated with an immune response to polyethylene glycol (PEG) during the first 4 exposure days to Jivi. A total of 35 patients with a median age of 8 years (range: 7 to 11) were enrolled and treated prophylactically for a minimum of 50 EDs and 26 weeks. The patients received a treatment regimen of Jivi twice weekly (40-60 IU/kg) at investigator’s discretion. Thirty-two patients completed the treatment phase and were offered continuation in an 18 month extension study. A key secondary endpoint was the annualized bleed rate (ABR)

PROTECT Kids Study was  a multi-center, prospective, single-arm trial to evaluate the pharmacokinetics, safety and efficacy of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients <12 years of age with severe hemophilia A. The primary efficacy endpoint was the annualized bleed rate (ABR).