+1-888-308-5802     
News Contact Us

AveXis presents data from ongoing Zolgensma trials in SMA patients

Author : Pankaj Singh | Published Date : 2019-05-06 
  • The incidence of SMA is one in every 10,000 live births.
  • SMA is often recognized as the leading genetic cause behind infant mortality.

AveXis, the Novartis-held biotechnology company based in the U.S., has reportedly declared data from the ongoing trials of Zolgensma (onasemnogene abeparvovec-xioi, AVXS-101) that exhibited positive results in a broad selection of patients afflicted with SMA (Spinal Muscular Atrophy).

Authentic sources close to the matter stated that the data includes the first presentation from the Phase 1 STRONG trial that exhibited motor function gains & milestone achievements through IT (intrathecal) delivery in patients with SMA Type 2; new data from Phase 3 STR1VE trial, which showed increase in motor functions and prolonged event-free survival, and first data presentation from the Phase 3 SPR1NT trial, that showed motor milestone achievement coupled with normal development in SMA patients who are treated pre-symptomatically. Sources further added that these data have been presented at the AAN (American Academy of Neurology) 2019 Annual Meeting.

SMA, for those uninitiated, is a critical neuromuscular disorder characterized by the loss of motor neurons resulting in progressive muscle weakness & paralysis. It generally occurs due to a defect in the SMN1 gene which codes SMN – a significant protein for survival of motor neurons.

According to David Lennon, President, AveXis, the company has witnessed Zolgensma offering speedy motor function improvement, prolonged survival and milestone achievements with a single, one-time dose. He went on to add that the robust data presented at the AAN signifies a growing body of evidence supporting Zolgensma’s usage as a potential, foundational therapy for the SMA treatment across a huge population.

Zolgensma is an investigational gene therapy which is in development at present as a one-time infusion for the SMA Type1. It was developed in close collaboration with Genethon, and in December 2018, the Food & Drug Administration (FDA) approved the Biologics License Application of the company for use of Zolgensma in in SMA Type 1 patients.

Source Credits: https://www.prnewswire.com/news-releases/avexis-presented-robust-data-at-aan-demonstrating-efficacy-of-zolgensma-in-broad-spectrum-of-spinal-muscular-atrophy-sma-patients-300843943.html

http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2397127

About Author

Pankaj Singh

Pankaj Singh

Endowed with a post graduate degree in management and finance, Pankaj Singh has been a part of the online content domain for quite a while. Having worked previously as a U.K. insurance underwriter for two years, he now writes articles for fractovia.org and other online portals. He can be contacted at- [email protected] | https://twitter.com/PankajSingh2605

Related News

Revival in UK hotel bookings after COVID-19 downturn, says Premier Inn

Published Date: 2020-07-09         Author: Pankaj Singh

UK has registered a revival in hotel bookings for conventional regional tourist destinations as people have started planning summer trips to the seaside and countryside, stated Premier Inn owner. The Chief Executive of Whitbread, Alison Brittain, has stated that larger cities like London continue... Read More

Fujitsu gravitates toward 'new normal' with new office working style

Published Date: 2020-07-08         Author: Pankaj Singh

Fujitsu Limited, a Japanese multinational IT company headquartered in Tokyo, has reportedly announced that it will boost its shift to becoming a digital transformation organization with an ambitious plan to transform the working style for its employees in Japan in the light of COVID-19 pandemic. ... Read More

Britain nears USD 625 million COVID-19 vaccine deals with Sanofi/GSK

Published Date: 2020-07-06         Author: Pankaj Singh

With over 100 vaccines are being tested and developed to stop the spread of COVID-19, it seems that governments worldwide are racing to secure supplies of vaccines even before their efficiency is proven. As per the latest news, Britain government is close to a USD 625 million supply deals with Sanof... Read More

© 2020 Fractovia. All Rights Reserved