Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy.

ABSTRACT:

Introduction

This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy.

Objective

We comprehensively assessed the safety of intravenous onasemnogene abeparvovec from preclinical studies, clinical studies, and postmarketing data.

Methods

Single-dose toxicity studies were performed in neonatal mice and juvenile or neonatal cynomolgus nonhuman primates (NHPs). Data presented are from a composite of preclinical studies, seven clinical trials, and postmarketing sources (clinical trials, n = 102 patients; postmarketing surveillance, n = 665 reported adverse event [AE] cases). In clinical trials, safety was assessed through AE monitoring, vital-sign a

SUBMITTER: Day JW 

PROVIDER: S-EPMC8473343 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature