Skip to main content
ZOLGENSMA
Proper Name
Onasemnogene Abeparvovec-xioi
Indication
For the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Description

ZOLGENSMA is a suspension of an adeno-associated viral vector-based gene therapy for intravenous infusion. It is a recombinant self-complementary AAV9 containing a transgene encoding the human survival motor neuron (SMN) protein, under the control of a cytomegalovirus enhancer/chicken-β-actin hybrid promoter.

Manufacturing Platform

PARAMETER

DATA

Manufacturer

AveXis, Inc

Transgene

Survival motor neuron gene (SMN1)

Indication

For the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Virus and Serotype

AAV serotype 9 (AAV9)

Cell Substrate

Human embryonic kidney cells (HEK293)

Manufacturing platform

Plasmid triple transfection

Dose in vial/final container

2.0 10e13 vector genomes / mL

Dose / patient

1.1 10e13 vector genomes / kg

SUPPORTING CLINICAL TRIALS

NCT

TRIAL PHASE

SUBJECTS ENROLLED

STUDY TITLE

COUNTRIES

Completed Phase 1 trial

NCT03421977

1

13

Long-term follow-up study for patients from AVXS-101-CL-101

United States

 Ongoing trials

NCT03306277

3

22

Gene replacement therapy clinical trial for participants with Spinal Muscular Atrophy type 1

United States

NCT03505099

3

30

Pre-symptomatic study of intravenous onasemnogene abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for patients with multiple copies of SMN2

United States, Italy, Australia, Belgium, Israel, Japan, Spain, Canada, Taiwan, Germany, Korea, Republic of, United Kingdom

NCT03461289

3

33

Single-dose gene replacement therapy clinical trial for patients with spinal muscular atrophy type 1

Belgium, France, Italy, United Kingdom

Key Regulatory Milestones

24/05/2019

US FDA Approval

05/18/2020

EU approval

12/16/2020

Health Canada approval

03/19/2020

Japan Approval
Advisory Committee

No advisory committee meeting was held because initial review of information submitted in the BLA did not raise concerns or controversial issues that would have benefited from an advisory committee discussion.

Advanced Facts