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Press release / May 05, 2019

AveXis presented robust data at AAN demonstrating efficacy of Zolgensma® in broad spectrum of spinal muscular atrophy (SMA) patients

Interim data reported for the first time from STRONG in SMA Type 2 showed rapid motor function gains and milestone achievements with intrathecal Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101…
Ad hoc release / May 24, 2019

AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)

SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2[1],[2…
Press release / Aug 07, 2019

Novartis stands behind Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Basel, August 6, 2019 – Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma® (onasemnogene abeparvovec-xioi). First and…
Press release / May 19, 2020

AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)

Zolgensma® (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with…
Ad hoc release / Mar 19, 2020

Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)

Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at…
Press release / Oct 01, 2020

Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function and milestone achievement

Nearly two-thirds of patients (65.6%) in STR1VE-EU have already achieved developmental motor milestones not observed in the natural history of SMA Type 1 at a mean duration of follow-up of 10.6…
Press release / Mar 24, 2020

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

Interim SPR1NT data showed presymptomatic babies with SMA treated with Zolgensma® (onasemnogene abeparvovec-xioi) soon after birth achieved age-appropriate motor milestones In addition to…
Press release / Sep 19, 2019

AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1

New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gainUpdated results from global…
Press release / Dec 03, 2018

Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

The AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)[1], filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative…
Press release / Apr 01, 2019

AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado

Facility to become the largest of four state-of-the-art sites involved in manufacturing of AveXis gene therapies for pipeline of rare genetic diseases including spinal muscular atrophy …