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Davide Matino at BIC 2025: Phase 3 Data on Fitelparvovec for Hemophilia A
Davide Matino/LinkedIn - First Author of The Article
Sep 15, 2025, 14:26

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”

Davide Matino at BIC 2025: Phase 3 Data on Fitelparvovec for Hemophilia A

More from BIC 2025 featured in Hemostasis Today.

AAV6 BIC 2025 Bleeding Episodes Davide Matino FVIII haemophilia A Health Hematology Hemostasis Hemostasis Today Medicine Pfizer Sangamo Therapeutics thrombosis thrombotic event
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