Efficacy and Safety of Giroctocogene Fitelparvovec in Adults With Moderately Severe to Severe Hemophilia Α

Highlights From the 66th ASH Annual Meeting and Exposition

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults With Moderately Severe to Severe Hemophilia Α: Primary Analysis Results From the Phase 3 ΑFFINE Gene Therapy Trial

Presenting Author: Steven W. Pipe, MD

Andrew D. Leavitt, MD¹, Kaan Kavakli^2*, Laurent Frenzel, MD, PhD^3*, Ali Bülent Antmen, MD, PhD^4*, Margareth Ozelo, MD⁵, Davide Matino, MD, MSc^6,7*, Hazza Alzahrani, MD^8*, Barbara A. Konkle, MD⁹, Steven W. Pipe, MD¹⁰, Jerome M. Teitel, MD¹¹, Li-Jung Tseng, PhD, MBA^12*, Annie F. Fang, MD, PhD^12*, Florence Ganne, PhD^13*, Gregory Dirusso, MD¹⁴, Jeremy Rupon, MD, PhD¹⁴, Pascal Klaus, MD^15*, Jasmine Healy, PhD^16*, Delphine Agathon, PhD^13*, Francesca Biondo, MD^17*, and Frank Plonski, MA, RN, BS^14*

Department of Medicine and Department of Laboratory Medicine, University of California San Francisco, San Francisco, CA
Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
Department of Haematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris, France
Department of Pediatric Hematology, Acibadem Adana Hospital, Adana, Turkey
Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
Thrombosis and Atherosclerosis Research Institute (TaARI), McMaster University, Hamilton, ON, Canada
Department of Medicine, McMaster University, Hamilton, ON, Canada
Adult Hematology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
Washington Center for Bleeding Disorders and the University of Washington, Seattle, WA
University of Michigan, Ann Arbor, MI
Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada

Data Points

AFFINE Phase 3

Figure 1: Annualized Bleeding Rate

The AFFINE Phase 3 study is an open-label, single-arm, multi-site trial, assessing the efficacy and safety of giroctogene fitelparvovec, an AAV6 gene therapy. Among the 50 patients treated, 64% (32/50) experienced no bleeding events from Week 12 through ≥ 15 months post-infusion, with a median follow-up of 33.6 months (range: 14.5—44.4). Over the total follow-up, 74 participants reported 624 adverse events (AEs), mostly mild or moderate with no deaths or discontinuations occurring. The most common treatment-related AEs were pyrexia, increased ALT, and headache. Elevated ALT was observed in 46.7% of participants, with 63% of participants requiring corticosteroids.

Overall, the therapy achieved durable FVIII expression, with significantly reducing bleeding rates compared to routine FVIII prophylaxis, and was well-tolerated with sustained efficacy across primary and secondary endpoints.