The U.S. Food and Drug Administration (FDA) has approved Jivi® for the treatment of severe hemophilia A in children aged 7 to 12 years. Hemophilia A is a condition where the blood does not clot properly due to a deficiency of Factor VIII. Jivi® is a long-acting medicine made using recombinant technology. It is manufactured by the Bayer pharmaceutical and biotechnology company.
In August 2018, the FDA initially approved Jivi® for use in hemophilia A individuals aged ≥12 years who had previously received treatment. The use of the medication for on-demand bleeding episode control and treatment, surgical bleeding management, and regular preventive treatment to lower the incidence of bleeding episodes was all covered by the approval.
Hemophilia affects around 400,000 people globally. It is an inherited disorder causing a lack or defect in blood clotting proteins. Hemophilia A is the most common type. It leads to frequent bleeding and potential joint damage. Based on the recent data, about 33,000 males in the U.S. are living with this condition. The FDA approval of Jivi® marks an important milestone and highlights Bayer’s ongoing commitment to providing effective treatment options for people with hemophilia A.
This FDA approval was based on the results from two studies: PROTECT Kids and Alfa-PROTECT. These studies showed that Jivi® was safe and effective in pediatric patients aged 7-12 years with severe hemophilia A. Both studies were prospective, multi-center, and single-arm trials.
The PROTECT kids study was conducted to assess the effectiveness, safety, and pharmacokinetics of Jivi® for preventing and treating bleeding in previously treated children aged under 12 years with severe hemophilia A. The primary efficacy outcome was the annualized bleed rate (ABR). Alfa-PROTECT was used to evaluate the safety of Jivi® for treatment and prophylaxis of bleeding in previously treated children aged from 7 to 12 years with severe hemophilia A. The study evaluated the risks of hypersensitivity and immune response to polyethylene glycol (PEG) during the first four treatments. A total of 35 patients (median age = 8 years) received twice-weekly Jivi® for at least 50 exposure days and 26 weeks. Thirty-two patients completed treatment and could join an 18-month extension. The secondary endpoint was ABR.
Jivi® is contraindicated in patients with hypersensitivity to PEG and animal proteins. Allergic reactions may be severe and require immediate discontinuation of treatment. The most common side effects (incidence ≥5%) include cough, fever, abdominal pain, and headache.
Jivi® is not approved for use in pediatric patients younger than 7 years due to a high risk of allergic reactions and reduced effectiveness. It is also not intended for patients who have never received treatment before, and for the treatment of von Willebrand disease.
The initial recommended dose of Jivi® for 7-12 years is 60 IU/kg twice weekly. For adults and adolescents, the dose is 30-40 IU/kg twice weekly or 45-60 IU/kg every 5 days with flexible dosing based on the bleeding episodes.
Bayer is committed to addressing the needs of the hemophilia A community. The FDA’s approval for children aged 7 to 12 years highlights our ongoing dedication to supporting patients and their families throughout their treatment journey,” said Jessica Charlet, Scientific Director, Bayer. This important milestone reinforces Bayer’s dedication to supporting individuals living with hemophilia A.
Reference: Business Wire. 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). Published May 1, 2025. Accessed May 21, 2025. 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)
