Davide Matino at BIC 2025: Phase 3 Data on Fitelparvovec for Hemophilia A
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
”Updated Ph3 data on gene therapy for haemophilia A with giroctocogene fitelparvovec (AAV6, 3e13 vg/kg in 74 participants), presented by Davide Matino at BIC meeting 2025:
~35% of participants maintained near-normal to normal FVIII activity (40–150%) at 2 years post-infusion
Durability data is impressive: sustained efficacy approaches three years of follow-up, demonstrating the transformative potential of this AAV6 approach
- Slow decline pattern:
– Year 1 mean: 51.7%
– Year 2 mean: 51.9% (stabilization)
– Year 3 mean: 40.7% (median: 38.0%)
Note: Only 8 participants had 3-year follow-up
- Sustained FVIII levels >5% in >80% of participants at 15 and at 24 months
- 98.3% reduction in treated bleeding episodes (4.08 → 0.07 ABR post-infusion)
- Transient FVIII activity >150% in nearly 50%, managed with low-dose prophylactic DOAC in 30%
- One participant developed a thrombotic event (history of thrombosis and risk factors)
- 2 participants resumed FVIII prophylaxis
Generally well-tolerated safety profile with manageable ALT elevations (ALT ↑ in 61%)
High rate of infusion-related reactions (75%), but no discontinuation of study drug administration
One case of new cancer at 35 months (oral SCC, not related to study drug); in total 2 malignancies reported
One case of FVIII inhibitor development (more to follow tomorrow!)
Despite these results, Pfizer has discontinued this program and terminated their collaboration with Sangamo Therapeutics in December 2024”
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