U.S. FDA Approves Pfizer’s BEQVEZ? for Hemophilia B

SciFocus/May 2024 -- The FDA greenlights 辉瑞 ’s revolutionary gene therapy BEQVEZ? for adults with moderate to severe Hemophilia B, marking a significant stride in personalized medicine. Here are the key highlights:

- BEQVEZ Efficacy: A one-time dose has shown significant reduction in bleeds post-treatment, with a median of zero bleeds after up to three years of follow-up.

- Patient Eligibility: Approved for adults with Hemophilia B who currently use FIX prophylaxis therapy, experience life-threatening hemorrhages, or have repeated serious spontaneous bleeding episodes without neutralizing antibodies to AAVRh74var capsid.

- Transformative Potential: BEQVEZ offers a life-changing alternative to regular FIX infusions, potentially sparing patients from treatment burdens and painful joint damage.

- Global Impact: With over 38,000 people worldwide living with Hemophilia B, BEQVEZ’s approval opens doors for better disease management and improved patient outcomes.

- Comprehensive Support: Pfizer’s innovative warranty program aims to provide financial protection and ensure durability of patient response to treatment.

- Clinical Study: Approval based on successful outcomes from the pivotal BENEGENE-2 study, showcasing BEQVEZ’s efficacy and safety profile over a follow-up period of up to 15 years.

Pfizer’s BEQVEZ heralds a new era in hemophilia treatment, offering hope and long-term benefits to eligible patients. #HemophiliaB #GeneTherapy #FDAApproval

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