AVXS 101

Drug Profile

AVXS 101

Alternative Names: AVXS-101; ChariSMA; rAAV9 - AveXis; scAAV9.CB.SMN; SMA-based therapeutic - AveXis; Spinal muscular atrophy gene therapy - AveXis

Latest Information Update: 22 May 2017

Price : $50

At a glance

  • Originator AveXis
  • Developer AveXis; Nationwide Children's Hospital
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Spinal muscular atrophy
  • New Molecular Entity No

Highest Development Phases

  • Phase I Spinal muscular atrophy

Most Recent Events

  • 25 Apr 2017 Updated efficacy data from a phase I trial in Spinal muscular atrophy type 1 released by AveXis
  • 18 Apr 2017 Pharmacodynamics and adverse events data from a preclinical trials in Spinal muscular atrophy released by AveXis
  • 16 Mar 2017 Interim adverse events and efficacy data from a phase I trial in Spinal muscular atrophy released by AveXis
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top