Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

September 26, 2024
Dennis Thompson HealthDay Reporter

A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.

People with hemophilia B saw their bleeding episodes drop by an average 71% following a single infusion of Beqvez (fidanacogene elaparvovec), researchers reported Sept. 25 in the New England Journal of Medicine.

Even better, more than half of the study’s 45 patients did not have any bleeds at all after receiving the gene therapy.

Hemophilia B is a rare inherited bleeding disorder that causes people to have lower levels of clotting factor IX, a blood protein that helps form clots.

The gene therapy uses a hollowed-out virus to deliver a working copy of the factor IX gene to a patient’s liver, enabling the body to start producing the clotting factor, researchers said.

“What we saw from patients in this study was that within a few days of receiving the gene therapy infusion, it took root, and their bodies started making factor IX for the first time in their lives,” said lead researcher Dr. Adam Cuker, clinical director of the Penn Blood Disorders Center and the Penn Comprehensive Hemophilia Program at the University of Pennsylvania.

“We always want to be careful about using the word ‘cure’ especially until we have longer follow-up data, but for many of these patients, it’s been life changing,” Cuker said in a UPenn news release.

Based on this clinical trial data, the U.S. Food and Drug Administration approved Beqvez in April 2024. The trial was paid for by Pfizer, which developed Beqvez.

Currently, people with hemophilia B must receive regular infusions of factor IX. Patients can require these infusions anywhere from once every two weeks up to several times a week.

By contrast, the new gene therapy only requires a single infusion dose. Most patients didn’t need to keep getting factor IX infusions after getting the gene therapy.

“We hear from people born with hemophilia that -- even if their disease is well-managed -- there’s this burden that’s always in the back of their mind. The frequent infusions, the cost of treatment, the need to plan for infusions when traveling, what happens if they do experience a bleed, and so on, is always there,” Cuker said.

“Now that we have patients who were treated in this study and are essentially cured of their hemophilia, they’re telling us about realizing a new, ‘hemophilia-free state of mind.’ As a physician, it’s amazing to see my patients so happy with their new reality,” Cuker added.

More information

The Cleveland Clinic has more about hemophilia B.

SOURCE: University of Pennsylvania, news release, Sept. 25, 2024

Gene Therapy Hemophilia