FDA approves Jivi for patients aged 7 years and older with hemophilia A | Image credit: Contemporary Pediatrics

On May 19, 2025, the FDA approved Jivi (antihemophilic factor [recombinant], PEGylated-aucl; Bayer) to use in pediatric patients aged 7 years and older with hemophilia A (congenital Factor VIII deficiency), according to a press release from Bayer.¹

Jivi is a recombinant DNA-derived, extended half-life factor VIII concentrate that was first approved by the FDA in August 2018 for use in previously treated patients aged 12 years and older with hemophilia A for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes.

According to Bayer, Jivi is not indicated for use in children younger than 7 years of age because of a greater risk for hypersensitivity reactions and/or loss of efficacy, previously untreated patients, or for the treatment of von Willebrand disease.

Jivi clinical trial information

The expanded approval of Jivi was based on data from the Alfa-PROTECT and PROTECT Kids studies, which demonstrated both safety and efficacy of the factor VIII concentrate in children aged 7 to younger than 12 years with severe hemophilia A.

In Alfa-PROTECT (NCT05147662)—a single-arm, multi-center, prospective study that evaluated the safety of Jivi infusions for the prophylaxis and treatment of bleeding—investigators 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.^1,2 In all, 35 patients with a median age of 8 years (7-11) were enrolled and treated prophylactically for a minimum of 50 EDs and 26 weeks. Patients received Jivi twice per week at the investigator's discretion (40-60 IU/kg). There were 32 patients who completed the treatment phase. These patients were offered continuation in an 18-month extension study. The PROTECT Kids study evaluated the pharmacokinetics, safety, and efficacy of Jivi, with the annualized bleed rate being the primary endpoint.¹

Hemophilia A is the most common type of hemophilia, in which blood clotting is impaired because there is a lack or defect of coagulation FVIII. Patients often repeatedly experience bleeds in muscles, joints, or other tissues, which can lead to chronic joint damage.

The company noted that Jivi is contraindicated in patients who have a history of hypersensitivity reactions to the active substance, polyethylene glycol (PEG), mouse or hamster proteins, or other constituents of the product. Patients should be monitored for hypersensitivity symptoms, as hypersensitivity reactions, including severe allergic reactions, have occurred with Jivi.

"Bayer remains dedicated to meeting the needs of the hemophilia A community," said Jessica Charlet, scientific director, Bayer, in a statement. "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."

References:

1. US FDA grants for Jivi® antihemophilic factor (recombinant), PEGylated-aucl in pediatric patients 7 to under 12 years of age with hemophilia A (congenital Factor VIII deficiency). Bayer. Press release. May 19, 2025. Accessed May 21, 2025. https://www.bayer.com/en/us/news-stories/jivi-for-hemophilia

2. 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). ClinicalTrials.gov. Updated April 25, 2025. Accessed May 21, 2025. https://clinicaltrials.gov/study/NCT05147662