Adam Cuker, MD, on Higher Efficacy of Gene Therapy Over SOC in Hemophilia B


The associate professor of medicine at University of Pennsylvania discussed new data from the BENEGENE-2 study of fidanacogene elaparvovec.

“I think fidanacogene elaparvovec is promising for a couple of reasons. We see that with a single infusion there was superiority to standard-of-care in reducing bleeds, and there were no worrisome safety signals that emerged. This appeared to be a durable response over 24 months, but of course, longer-term follow-up is needed. We hope for, essentially, a cure for hemophilia with gene therapy. And based on these results, preliminary though they are, they're a very encouragingstep toward that goal.”

A single infusion treatment of fidanacogeneelaparvovec demonstrated superiority over standard-of-care factor IX therapy in reducing annualized bleeding rates (ABR) in patients with hemophilia B, according to updated data from Pfizer’s BENEGENE-2 phase 3 clinical trial (NCT03861273). Compared with a 6-month lead-in period, patients treated with the therapy had a 78% reduction in ABR for treated bleeds (P = .0001)and a 92% reduction in the annualized infusion rate of exogenous FIX following infusion with fidanacogene elaparvovec (P <.0001).

CGTLive spoke with study investigator Adam Cuker, MD, associate professor of medicine, University of Pennsylvania, to learn more about BENEGENE-2 and the new superiority data. He also touched on the positive durability data seen in patients treated with fidanacogeneelaparvovec.

Pfizer announces positive top-line results from phase 3 study of hemophilia B gene therapy candidate. News release. Pfizer. December 20, 2023.
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