Why BEQVEZ?
BEQVEZ is a one-time gene therapy that offers a chance of bleed protection without the need for factor IX prophylaxis (FIX prophy)
How was BEQVEZ studied?
The main BEQVEZ study:
- 45 participants were enrolled in an ongoing study
- Before the BEQVEZ infusion, all had at least 6 months of routine FIX prophy
- The study compared the number of bleeds participants experienced following the BEQVEZ infusion versus the bleed rate with routine FIX prophy before the BEQVEZ infusion (the lead-in period)†
- This ongoing study aims to follow each participant’s results for up to 6 YEARS
In the main study, BEQVEZ helped participants achieve:
Participants had a 44% decrease in the number of bleeds per year on BEQVEZ vs routine FIX prophy
In the efficacy evaluation period, participants experienced an average of 2.5 bleeds per year following BEQVEZ, compared to 4.5 while they were on routine FIX prophy during the lead-in period.
60% of participants had ZERO bleeds during the efficacy evaluation period (27 out of 45)
While on routine FIX prophy, 29% of participants (13 out of 45) had zero bleeds in the lead-in period.
87% of participants were prophy free during the efficacy evaluation period (39 out of 45 participants)
13% of participants (6 out of 45) returned to FIX prophy from 0.4 to 1.7 years after the BEQVEZ infusion.
Safety studied since 2017: The BEQVEZ safety profile was studied in the longest-running clinical development program of any approved gene therapy for hemophilia B
The most common side effect seen in ≥5% of participants following treatment with BEQVEZ was raised liver enzymes
- 53.3% of participants (24 out of 45) in the main BEQVEZ study
- 62% (28 out of 45) received corticosteroids for raised liver enzymes and/or decrease in FIX activity‡
- 13.3% of participants (2 out of 15) in the initial BEQVEZ studies
- 20% (3 out of 15) received corticosteroids for raised liver enzymes and/or decrease in FIX activity‡
These are not all the possible side effects of BEQVEZ. Your care team will monitor you for possible infusion reactions on the day of your infusion.
Your healthcare professional will monitor liver enzymes and FIX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes.
Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache.
