FDA Extends Approval of Jivi to Younger Hemophilia A Patients

The FDA has approved Bayer’s Jivi for use in children 7 years of age and older with hemophilia A, a bleeding disorder that is caused by a lack or low level of clotting factor VIII. Hemophilia A is caused by mutations in the F8 gene, according to the CDC.

Jivi is a recombinant DNA-derived, extended half-life factor VIII concentrate and is already approved to treat patients 12 years of age and older with hemophilia A. Jivi was engineered with polyethylene glycol (PEG), which increases FVIII’s stability and allows for less frequent dosing.

The recommended initial dosage of Jivi for both adults and adolescents is 30-40 IU/kg twice weekly with the ability to dose every five days. The dosing can be adjusted based on bleeding episodes.

Jivi is 100% covered under the Medicare medical benefit nationally, according to the product website. Bayer offers a free trial program regardless of insurance type. Commercially insured patients can receive Jivi for a $0 copay, with a limit of $20,000 a year.

The approval for children 7 to 11 years of age 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.

The Alfa-PROTECT study was a single-arm trial 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 enrolled 35 patients who received Jivi twice weekly at the 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.

The PROTECT study was a 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.

Additionally, Bayer published the fourth interim data in 2023 from the ongoing HEM-POWR study, which aims to provide a real-world assessment of Jivi using patient e-diaries and physician records.

As of Aug. 1, 2023, 339 patients were enrolled in the study; the safety analysis included data from 332 patients, including women and those with moderate disease, which were not included in the Jivi label. The full set analysis includes 227 patients and also includes data from male patients with several hemophilia.

This analysis found that the total annualized bleed rate for the severe subgroup during the 12 months before Jivi was 3.2 and during the observation period with Jivi was 1.8. In this subgroup, 46% of patients had no bleeding episodes during the observation period

The safety analysis of 332 patients found that there were just two cases of drug-related treatment-emergent adverse events (TEAE), and one case of a drug-related serious TEAE. There was one case of tendonitis, a non-drug-related adverse event, that led to the continuation of Jivi.