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.
|
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 |
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 |
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 |
|
24/05/2019 |
|
|
05/18/2020 |
|
|
12/16/2020 |
|
|
03/19/2020 |
Japan Approval |
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.