FDA Approved Antihemophilic factor, PEGylated-Aucl For Pediatric Patients With Hemophilia A

Antihemophilic factor (recombinant), PEGylated-aucl (Jivi; Bayer) received FDA approval for treatment of pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency). The decision was based on data from the Alfa-PROTECT (NCT05147662) and PROTECT Kids (NCT01775618) trials.^1,2

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Hemophilia A occurs in approximately 1 in 5617 live male births and affects an estimated 30,000 males in the US, according to the CDC. Of these cases, over half of patients have the severe form. Hemophilia A, also called factor VIII (8) deficiency, is a genetic disorder caused by missing or defective key protein needed for blood clotting, factor VIII (FVIII).³

Jivi is a recombinant DNA-derived, extended half-life factor VIII concentrate that was originally approved by the FDA in 2018 for pediatric patients ages 12 and older for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes. However, this approval expands the indication to include children as young as 7 years old.⁴

The safety and efficacy of Jivi were determined in the Alfa-PROTECT and PROTECT Kids studies. Alfa-PROTECT is a multicenter, prospective, single-arm study that evaluated the safety of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients aged 7 to less than 12 years with severe hemophilia A. The trial’s primary focus was on assessing the potential risk of hypersensitivity and loss of drug efficacy due to an immune response to polyethylene glycol during the first 4 exposure days.⁴

A total of 35 patients (median age: 8 years; range: 7 to 11) were enrolled and received prophylactic treatment with Jivi twice weekly (40–60 IU/kg), as determined by the investigator, for at least 50 exposure days over a minimum of 26 weeks. Thirty-two patients completed the treatment phase and were offered participation in an 18-month extension study. A key secondary end point was the annualized bleed rate (ABR).⁴

PROTECT Kids was a multicenter, prospective, single-arm trial to evaluate the pharmacokinetics, safety, and efficacy of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients under 12 years of age with severe hemophilia A. The primary efficacy end point for this trial was ABR.⁴

The expanded FDA approval of Jivi represents a meaningful advancement in the treatment of pediatric patients with severe hemophilia A, offering a safe and effective prophylactic option for children as young as 7 years old.

REFERENCES

1. A study to learn how safe the study treatment BAY94-9027 is and how it affects the body in previously treated children aged 7 to less than 12 years with severe hemophilia a, a genetic bleeding disorder that is caused by the lack of a protein called clotting factor 8 (FVIII) in the blood (Alfa-PROTECT). Updated April 25, 2025. Accessed May 19, 2025. https://clinicaltrials.gov/study/NCT05147662?rank=1

2. Safety and efficacy of BAY94-9027 in previously treated male children with haemophilia a. Updated August 21, 2020. Accessed May 19, 2025. https://clinicaltrials.gov/study/NCT01775618

3. Hemophilia A. National Bleeding Disorders Foundation. Accessed May 19, 2025. https://www.bleeding.org/bleeding-disorders-a-z/types/hemophilia-a

4. U.S. FDA grants approval for Jivi® antihemophilic factor (recombinant), PEGylated-aucl in pediatric patients 7 to under 12 years of age with hemophilia A (Congenital Factor VIII Deficiency). Business Wire. May 19, 2025. Accessed May 19, 2025. https://www.businesswire.com/news/home/20250519742201/en/U.S.-FDA-Grants-Approval-for-Jivi-Antihemophilic-Factor-Recombinant-PEGylated-aucl-in-Pediatric-Patients-7-to-Under-12-Years-of-Age-With-Hemophilia-A-Congenital-Factor-VIII-Deficiency