FDA Approves Jivi for Pediatric Patients Aged 7 to 12 Years With Hemophilia A

Treatment options for children with hemophilia A have expanded with the FDA approval of Bayer's Jivi antihemophilic factor (recombinant), PEGylated-aucl for the treatment of pediatric patients between 7 and 12 years of age.¹

Hemophilia diagnosis is typically confirmed through specialized blood testing shortly after birth, and in some cases, prenatal genetic testing can detect hemophilia before delivery. | Image credit: Denira - stock.adobe.com

Previously, Jivi was approved in September 2018 for adults and adolescents aged 12 years and older with this hemophilia subtype.²

According to data collected from federally funded hemophilia treatment centers between 2012 and 2018, an estimated 33,000 males in the US are living with the disorder.³

Hemophilia A, which results from low levels of clotting factor VIII, is 3 to 4 times more common than hemophilia B, which is caused by low levels of clotting factor IX.⁴ Most individuals with hemophilia in the US are diagnosed at an early age. CDC data show that the median ages at diagnosis are:

• 1 month for severe hemophilia
• 8 months for moderate hemophilia
• 36 months for mild hemophilia

The CDC also reports that approximately two-thirds of infants diagnosed with hemophilia have a known family history of the condition. Diagnosis is typically confirmed through specialized blood testing shortly after birth, and in some cases, prenatal genetic testing can detect hemophilia before delivery.

Jivi can be used as on-demand treatment and for control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes.¹

Bayer stipulated that Jivi is still not indicated for use in children under 7 years of age due to a higher risk of hypersensitivity reactions and/or loss of efficacy. It is also not approved for use in previously untreated patients or for the treatment of von Willebrand disease.

"Bayer remains dedicated to meeting the needs of the hemophilia A community," stated Jessica Charlet, scientific director at Bayer, in a statement.¹ “The FDA’s approval for pediatric patients 7 to <12 years of age is reflective of Bayer’s commitment to the hemophilia A community and underscores our commitment to supporting families through their treatment journey.”

The recommended starting dose for adults and adolescents is 30 to 40 IU/kg administered twice weekly. Depending on individual response and bleeding patterns, the dosing interval may be extended to every 5 days at a dose of 45 to 60 IU/kg. Further adjustments—either to more or less frequent dosing—can be made based on the patient's bleeding episodes and clinical needs.

The approval was based on data from both the Alfa-PROTECT (NCT05147662) and PROTECT Kids (NCT01775618) studies, which collectively demonstrated the safety and efficacy of Jivi in pediatric patients with severe hemophilia A.

The Alfa-PROTECT study was a multicenter, prospective, single-arm trial that evaluated the safety of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients aged 7 to under 12 years. The study specifically focused on assessing the potential risk of hypersensitivity reactions and loss of efficacy associated with an immune response to polyethylene glycol (PEG) during the first 4 exposure days.

A total of 35 patients (median age 8 years, range 7–11) received prophylactic treatment with Jivi twice weekly (40–60 IU/kg) for at least 50 exposure days over a 26-week period. Thirty-two patients completed the treatment phase and continued in an 18-month extension study. The study met its objectives, with no unexpected safety concerns and favorable outcomes, including low annualized bleed rates (ABRs).

The PROTECT Kids study was a multicenter, prospective, single-arm trial that further evaluated the pharmacokinetics, safety, and efficacy of Jivi in previously treated pediatric patients under 12 years of age. The primary efficacy end point was the ABR, and the study successfully met this endpoint, demonstrating that Jivi effectively reduced bleeding episodes in this population. Safety findings were consistent with the known profile of Jivi.

References

1. 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). Press release. Bayer; 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

2. Joszt L. FDA approves new drug for adults and adolescents with hemophilia A. September 2, 2018. Accessed May 19, 2025. https://www.ajmc.com/view/fda-approves-new-drug-for-adults-and-adolescents-with-hemophilia-a

3. Soucie JM, Miller CH, Dupervil B, Le B, Buckner TW. Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia. 2020; 26:487-493. doi:10.1111/hae.13998

4. Data and statistics on hemophilia. CDC. May 15, 2024. Accessed May 19, 2025. https://www.cdc.gov/hemophilia/data-research/index.html