Pfizer Inc. announced that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. A Biologics License Application and European Marketing Authorization Application for marstacimab are currently under review with the FDA and EMA, respectively. BEQVEZ is an adeno-associated virus (AAV)-based gene therapy designed to introduce in the transduced cells a functional copy of the FIX gene encoding a high-activity FIX variant.

For eligible patients living with hemophilia B, the goal of this gene therapy is to enable them to produce FIX themselves via this one-time treatment rather than having to receive frequent infusions of FIX, as is the current standard of care. It is currently approved in the U.S. for the treatment of adults withmoderate to severe hemophilia B (congenital factor IX deficiency) who: Currently use factor IX prophylaxis therapy,or Have current or historical life-threatening bleeding episodes, or have repeated, seriousaneous bleeding episodes, and, Do not have neutralizing antibodies to Adeno-associated virus serotypeRh74var (AAVRh 74var) capsid as detected from an FDA-approved test. For eligible patients who are prescribed BEQVEZ, Pfizer offers personalized patient support services including financial assistance resources and logistical support through the Pfizer Gene Together program.

Eligible study participants have completed a minimum of six months of routine FIX prophylaxis therapy during the lead-in study (NCT03587116) and received a single intravenous infusion of BEQVEZ at a dose of 5 x 1011 vg/kg of body weight. Clinical trial participants will be followed for up to a total of 15 years, including six years in the BENEGENE-2 study and an additional nine years as part of a separate Phase 3 study (NCT05568719) to learn about the long-term safety and efficacy of BEQVEZ. R risks and uncertainties regarding the commercial success of BEQVEZ; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from clinical studies; whether and when drug applications may be filed in particular jurisdictions for BEQVEZ or any other product candidates; whether and when any applications that may be pending or filed for BEQVEZ and any other product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether BEQVEZ or any such other product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of BEQVEZ or any Other product candidates; uncertainties regarding the impact of COVID-19 on Pfizer's COVID-19 on the Pfizer's product candidates; uncertainties regarding the effect of COVID-19 onPizer's COVID-19.