2019 Press Releases

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05/24/19AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families
One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA   Annualized cost of Zolgensma is USD 425,000 per year for 5 years: 50% less than multiple established value-based pricing benchmarks including the 10-year current cost of chronic SMA therapy[1]   AveXis is working closely with payers to create 5-year outcomes-based agreements and novel pay-over-time options; Time is Neurons progra... 
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05/24/19AveXis 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]   Zolgensma (onasemnogene abeparvovec-xioi) is approved for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) including those who are pre-symptomatic at diagnosis   Zolgensma is designed to address the genetic root cause of SMA by replacing the... 
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05/07/19New AveXis data at AAN showed long-term durability of Zolgensma® in patients with spinal muscular atrophy (SMA) Type 1
Interim long-term follow-up data showed all enrolled Cohort 2 patients maintained motor function and milestones achieved during the Phase 1 START trial Mean age of follow-up since dosing with Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) was nearly four years, with some patients nearing five years of age  No loss of milestones or waning of effect in long-term follow-up of START adds to evidence of long-term durability of Zolgensma   Basel, Ma... 
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05/05/19AveXis 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)- New interim data from STR1VE in SMA Type 1 continued to show prolonged event-free survival, increases in motor function and significant milestone achievement consistent with Phase 1 START trial- Interim data reported for the first time from SPR1NT in pre-symptomatic SMA showed age-appropriate ... 
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04/16/19AveXis data reinforce effectiveness of Zolgensma® in treating spinal muscular atrophy (SMA) Type 1
  Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial  First-in-human biodistribution data show transduction in intended CNS targets and widespread SMN expression comparable to tissue from unaffected control   More than 150 patients treated with Zolgensma, only 5% of screened patients up to 5 years old excluded due to AAV9 antibody titers greater than 1:50Base... 
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04/01/19AveXis 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 atrophyAveXis plans to offer positions to all approximately 150 employees previously employed at the site, and to announce further expansion of new jobs in the near termInvestment in the Longmont campus adds to existing $115 million investment in Durham facility, leading to creation of more than 1,000 US-based, high-tech,... 
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