Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A.

ABSTRACT:

Introduction

Valoctocogene roxaparvovec is an investigational AAV5-based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A.

Aim

To report safety, tolerability, efficacy, and quality of life (QOL) among participants who received valoctocogene roxaparvovec in a phase 1/2 clinical study (NCT02576795).

Methods

Men ≥18 years of age with severe haemophilia A (FVIII ≤1 IU/dl) without history of FVIII inhibitors or anti-AAV5 antibodies received a single infusion of valoctocogene roxaparvovec and were followed for 5 years (6 × 10¹³ vg/kg dose, n = 7) and 4 years (4 × 10¹³ vg/kg dose, n = 6).

Results

Over the past 2 years, few adverse events and no FVIII inhibitors were reported. Pe

SUBMITTER: Pasi KJ 

PROVIDER: S-EPMC9291073 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature