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Today, Novartis' SMA gene therapy clinical application in China has been accepted, and the acquisition of the company for US$8.7 billion has been obtained!

 Last Update: 2021-11-04
 Source: Internet
 Author: User

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On October 21, the latest announcement by the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China showed that Novartis had submitted a clinical trial application for OAV101 injection (Zolgensma) in China and was accepted
.

This is a gene therapy developed by AveXis.

The first gene therapy for spinal muscular atrophy (SMA) approved by the US FDA

Screenshot source: CDE official website

SMA is a serious neuromuscular disease
.

It is a serious neuromuscular disease caused by a mutation in the SMN1 gene encoding Survival Motor Nervous Protein (SMN), resulting in the loss of SMN protein levels

Severe neuromuscular disease caused by a mutation in the SMN1 gene encoding Survival Motor Nervous Protein (SMN), resulting in the loss of SMN protein levels

Zolgensma (OAV101, or AVXS-101) was originally AveXis' main gene therapy candidate
.

It loads the transgene that normally expresses SMN protein into the AAV9 viral vector, and improves the transgene to improve its ability to produce SMN protein

It is designed to allow patients to express SMN protein in their cells for a long time after receiving a treatment, so as to achieve a "curative" effect

In May 2019, the therapy was approved for marketing in the United States

In terms of clinical efficacy, according to data published earlier by AveXis, in the pivotal clinical trial STR1VE-US, not only 91% of patients with severe type 1 SMA treated with Zolgensma reached the primary endpoint of event-free survival , but 22 patients in nine reached the more stringent "grow" standard
.

In the START clinical trial to test the long-term efficacy of Zolgensma, all patients are still alive as of March 2020 and do not require permanent respiratory assistance.

91% reached the primary endpoint of event-free survival; 9 reached the more stringent "thriving" criteria.

The results of another clinical trial called SPR1NT showed that using Zolgensma when children with SMA have not yet shown symptoms of disease can significantly improve the patient's exercise capacity
.

Among the 14 treated patients, 100% of the children reached the primary endpoint of sitting up independently for more than 30 seconds

The use of Zolgensma when children with SMA have not yet shown symptoms of disease can significantly improve the patient’s exercise capacity

What cannot be ignored is that gene therapy, as a revolutionary treatment, also has potential side effects and safety issues
.

For example, a study published in the New England Journal of Medicine showed that in a small number of patients treated with Zolgensma, side effects such as a significant increase in liver enzyme levels have also occurred

AveXis said in a press release that Zolgensma's clinical trial evidence shows that it can not only save the lives of patients, but also allow some patients to thrive as healthy children
.

After receiving one treatment, the efficacy of Zolgensma can be maintained for more than 5 years

Reference materials:

[1] The official website of the Center for Drug Evaluation of China National Medical Products Administration.
Retrieved Oct 21, 2021, from https:// The official website of the Center for Drug Evaluation of China National Medical Products Administration.
Retrieved Oct 21, 2021, from https:// [2]AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)https:// /en/AveXis-receives-FDA-approval-for-Zolgensma-the-first-and-only-gene-therapy-for-pediatric-patients-with-spinal-muscular-atrophy-SMA.
html

[2]AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)https:// /en/AveXis-receives-FDA-approval-for-Zolgensma-the-first-and-only-gene-therapy-for-pediatric-patients-with-spinal-muscular-atrophy-SMA.
html

[3] Novartis delivered strong Q2 performance, driven by momentum of key growth brands.
FY 2021 guidance unchanged.
Retrieved Jul 21, 2021, from https:// -q2-performance-driven-momentum-key-growth-brands-fy-2021-guidance-unchanged

[4] Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing, Retrieved March 24, 2020, from https://www.
novartis.
com/news/media-releases/zolgensma-data-shows-rapid-significant-clinically-meaningful-benefit-sma-including-prolonged-event-free-survival-motor-milestone-achievement-and-durability-now[ 5] AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2, Retrieved March 24, 2020, from https:// releases/avexis-presents-avxs-101-it-data-demonstrating-remarkable-increases-hfmse-scores-and-consistent-clinically-meaningful-response-older-patients-sma-type-2

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