Gene therapy Hemgenix given to 1st hemophilia B patient in Nevada
Male patient, 39, will be monitored regularly to ensure treatment’s success
A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B.
The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding and clotting clinic, and Joseph Lasky, MD, medical director of the foundation and its medical team. The patient will be monitored on a regular basis to ensure the treatment’s success.
“Being approved to provide this life-changing treatment is another example of how Cure 4 The Kids Foundation is bringing innovation and cutting-edge therapies to the patients of Nevada and the western United States,” Annette Logan-Parker, the foundation’s chief innovation and advocacy officer, said in a press release.
Hemgenix widely approved
The gene therapy, which was originally developed by uniQure, is approved in the U.S., Europe, and other regions. In the U.S., Hemgenix is approved for adults who are on standard clotting factor preventive treatment, are experiencing or have a history of life-threatening bleeding episodes, or have had serious recurrent bleeding episodes occurring spontaneously.
Hemophilia is a genetic disorder marked by the lack or deficiency in certain blood clotting factors that are necessary to prevent excessive bleeding. Hemophilia B, the second most common type of hemophilia, is caused by mutations in the F9 gene, which provides instructions for making a blood clotting protein called factor IX (FIX).
Before gene therapies, the mainstay option for managing hemophilia B was the use of prophylactic (preventive) FIX replacement therapies, which work to provide patients with the clotting factor they are missing. However, this requires patients to follow strict regimens, and does not eliminate the risk of spontaneous bleeds.
Hemgenix, administered as a single into-the-vein, or intravenous, infusion, delivers a working version of the F9 gene, called FIX-Padua, to patient cells. The therapy uses a modified, harmless virus as a vehicle to deliver the gene to liver cells, where most blood clotting factors are produced. The therapy’s ultimate goal is to restore the body’s ability to produce a functional version of FIX, so as to effectively prevent and control bleeds.
‘Ground-breaking’ Hemgenix gives patients freedom from routine infusions
“This treatment is ground-breaking,” Logan-Parker said, noting it “gives the patient a freedom from daily or weekly factor infusions they have not had since their original hemophilia diagnosis.”
Hemgenix’s approval was supported by early data from the Phase 3 HOPE-B trial (NCT03569891), which tested the therapy in people with severe or moderately severe hemophilia B.
The therapy, which was deemed to be safe, led to a reduction of 64% in the number of annual bleeds after two years of treatment. Also, most patients (94%) no longer required routine replacement therapies. More recent data showed both bleeding rates and the use of replacement therapies remained low for at least three years after Hemgenix treatment.
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