AVXS 101

Drug Profile

AVXS 101

Alternative Names: AVXS-101; ChariSMA; scAAV9.CB.SMN

Latest Information Update: 03 Apr 2018

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At a glance

  • Originator AveXis
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 1 protein expression stimulants
  • 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

  • 09 Apr 2018 AVXS 101 is expected to be launched for Spinal muscular atrophy in USA in 2019
  • 27 Mar 2018 AVXS 101 receives SAKIGAKE designation for the treatment of Spinal muscular atrophy type 1 in Japan
  • 13 Mar 2018 AveXis inn-licenses patents for delivery of AAV9 vector from Genethon
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