gene therapy

In a notable advancement for hemophilia treatment, Pfizer Inc. (NYSE: PFE) has received U.S. Food and Drug Administration approval for BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy aimed at adults suffering from moderate to severe hemophilia B. This innovative treatment could significantly alter the treatment landscape for patients traditionally reliant on regular factor IX (FIX) prophylaxis.

 

A Potential Game-Changer in Hemophilia Care

BEQVEZ is tailored for individuals who are on routine FIX prophylaxis, have a history of life-threatening bleeding, or experience recurrent serious spontaneous bleeding episodes, and have no neutralizing antibodies to the AAVRh74var capsid, as detected by an FDA-approved test.

According to Dr. Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program, BEQVEZ represents a potential paradigm shift, offering patients the possibility to significantly reduce their long-term medical and treatment burdens.

 

Efficacy Demonstrated in Clinical Trials

The FDA endorsement follows compelling evidence from the pivotal BENEGENE-2 study. This Phase 3 trial revealed that BEQVEZ could lower the annualized rate of bleeding significantly in comparison to traditional prophylaxis, with some patients experiencing zero bleeds over three years of follow-up.

 

Global Implications and Next Steps

Following its approval in Canada and the ongoing review by the European Medicines Agency (EMA), BEQVEZ has the potential to impact over 38,000 individuals worldwide living with hemophilia B. This therapy promises to ease the constant treatment regimen associated with the disease.

Source:
https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-beqveztm-fidanacogene-elaparvovec
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