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: 16 Oct 2017

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At a glance

  • Originator AveXis
  • 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 III Spinal muscular atrophy

Most Recent Events

  • 29 Sep 2017 Phase-III clinical trials in Spinal muscular atrophy (In infants, In neonates) in USA (IV) (NCT03306277)
  • 25 Apr 2017 Updated efficacy data from a phase I trial in Spinal muscular atrophy type 1 released by AveXis
  • 21 Apr 2017 Pharmacodynamics data from a preclinical trial in Spinal muscular atrophy presented at the 69th Annual Meeting of the American Academy of Neurology (AAN-2017)
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