One infusion of etranacogene dezaparvovec noninferior and superior to factor IX prophylaxis<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b><\/p>\n
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FRIDAY, March 3, 2023 (HealthDay News) — For patients with hemophilia B, one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec) is noninferior and superior to prophylaxis with factor IX, according to a study published in the Feb. 23 issue of the New England Journal of Medicine<\/em>.<\/p>\n Steven W. Pipe, M.D., from the University of Michigan in Ann Arbor, and colleagues conducted an open-label phase 3 study involving 54 men with hemophilia B. After a lead-in period (six months or longer) of factor IX prophylaxis, one infusion of etranacogene dezaparvovec was administered, regardless of preexisting AAV5 neutralizing antibodies. The annualized bleeding rate was assessed in a noninferiority analysis, which compared the rate during months 7 through 18 after etranacogene dezaparvovec treatment to the rate during the lead-in period.<\/p>\n The researchers found that from the lead-in period to months 7 through 18 after treatment, there was a decrease in the annualized bleeding rate from 4.19 to 1.51, for a rate ratio of 0.36, indicating noninferiority and superiority of etranacogene dezaparvovec versus factor IX prophylaxis. Factor IX activity increased from baseline by a least-squares mean of 36.2 and 34.3 percentage points at six and 18 months after treatment; posttreatment, use of factor IX concentrate decreased by a mean of 248,825 IU per year per participant. Participants with predose AAV5 neutralizing antibody titers of less than 700 had benefits and safety.<\/p>\n “Our findings suggest that gene therapy may reduce the burden of care and improve quality of life in patients with hemophilia B,” the authors write.<\/p>\n Several authors disclosed financial ties to pharmaceutical companies, including CSL Behring, which manufactures etranacogene dezaparvovec and funded the study.<\/p>\n Abstract\/Full Text (subscription or payment may be required)<\/a><\/p>\n <\/i><\/p>\n