Jivi approved by FDA for children with hemophilia A as young as 7
Treatment had previously been authorized for patients ages 12 and older
Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old.
Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer, is indicated to be used as an on-demand treatment to control active bleeds, as a perioperative therapy to manage bleeds during surgery, and as a routine prophylactic (preventive) therapy to reduce the frequency of bleeding episodes.
The therapy should not be used in children younger than 7 due to the increased risk of hypersensitivity reactions and/or loss of efficacy. It also should not be used in previously untreated patients.
The new approval “is reflective of Bayer’s commitment to the hemophilia A community and underscores our commitment to supporting families through their treatment journey,” Jessica Charlet, PhD, scientific director of Bayer, said in a company press release.
Decision to expand approval based on data from earlier trials
Hemophilia A is caused by mutations that lead to a deficiency in factor VIII (FVIII), a protein that helps the blood to clot. Replacement therapies are a cornerstone of hemophilia A treatment and involve administering a functional version of FVIII to restore blood clotting, making up for the missing clotting factor.
The naturally occurring version of the FVIII protein is relatively unstable, so it doesn’t last long when administered into the body. Jivi is an extended half-life replacement therapy, where the FVIII protein has been modified to be more stable and last longer in the body, allowing the therapy to be given less frequently. The version of FVIII in Jivi has specifically been modified with a chemical process called PEGylation, which involves the attachment of a nontoxic molecule called polyethylene glycol, or PEG, that makes FVIII less likely to be broken down and more long-lasting.
The therapy is administered via an into-the-vein infusion. When used for routine prophylaxis, it is given twice weekly.
The FDA initially approved Jivi in 2018 for people with hemophilia A ages 12 and older.
The decision to expand its approval to children as young as 7 was based in part on data from a Phase 3 clinical trial called PROTECT VIII Kids (NCT01775618), which tested the therapy in children younger than 12 with hemophilia A who had previously been on other FVIII replacement treatments. Data from another Phase 3 trial called Alfa-PROTECT (NCT05147662), which tested Jivi in previously treated children ages 7 to 11, also supported the approval.
Pooled data from the two studies showed children ages 7 to 11 receiving prophylactic treatment with Jivi twice weekly experienced less than two bleeds per year. On-demand treatment with Jivi was also effective for managing most bleeds that did occur, with 97% of the bleeding episodes being successfully treated with one or two infusions.
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