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Lancet Neurol: How effective and safe is gene replacement therapy for infant symptomatic spinal muscular atrophy type 1?

 Last Update: 2021-11-02
 Source: Internet
 Author: User

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Spinal muscular atrophy is a rare autosomal recessive neurological disease.
It is caused by the biallelic loss of function of the survival motor neuron 1 (SMN1) gene, which leads to motor neuron dysfunction and degeneration
.

Although the similar SMN2 gene is preserved in spinal muscular atrophy, a full-length SMN protein produced by SMN2 alone is not enough

Spinal muscular atrophy is a rare autosomal recessive neurological disease.

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The effectiveness of SMN targeted therapy significantly improves the prognosis of patients
.

Nusinesen US Food and Drug in December 2016 Management Board ( the FDA approved), and in June 2017 approved by the European Medicines Agency (European Medicines Agency), and is outside one kind of intrathecal injection of SMN2 exon 7 Antisense oligonucleotide modulator of splicing

Manage FDA

Onasemnogene abeparvovec (formerly A VXS-101) 10 was approved by the FDA in May 2019 and conditional EMA approval in May 2020.
It is a gene replacement therapy designed to target the genetic causes of spinal muscular atrophy
.

With only one intravenous infusion, onasemnogene abeparvovec can deliver human SMN through a self-complementary adeno-associated virus serotype 9 (AA V9) vector, which is driven by a mixed cytomegalovirus enhancer-chicken β-actin promoter.

Onasemnogene abeparvovec (formerly A VXS-101) 10 was approved by the FDA in May 2019 and conditional EMA approval in May 2020.

STR1VE-EU is a multi-center, single-arm, single-dose, open-label Phase 3 trial in Italy (n=4), the United Kingdom (n=2), Belgium (n=2) and France (n=1) Of 9 locations (hospitals and universities)
.

The study recruited patients with type 1 spinal muscular atrophy younger than 6 months (180 days) and common biallelic pathogenic SMN1 exon 7-8 deletions or point mutations, and one or two copies of SMN2

Multicenter, single arm, single dose, open labelMulticenter, single arm, single dose, open label

From August 16, 2018 to September 11, 2020, 41 patients with spinal muscular atrophy underwent eligibility assessment
.

The median age of using onasemnogene abeparvovec was 4.

STR1VE-EU shows the effect of onasemnogene abeparvovec on infants with type 1 symptomatic spinal muscular atrophy

Mercuri E, Muntoni F, Baranello G, et al.

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