Onasemnogene abeparvovec - AveXis

Drug Profile

Onasemnogene abeparvovec - AveXis

Alternative Names: AAV9-CBA-SMN1-gene-therapy-AveXis; Adeno-associated-serotype-9-chicken-beta-actin-survival-motor-neuron-gene-therapy-AveXis; AVXS 101; ChariSMA; scAAV9.CB.SMN; SMA1-gene-therapy-AveXis; SMN1-gene-therapy-AveXis; SMNT-gene-therapy-AveXis; Spinal-muscular-atrophy-gene-therapy-AveXis; Survival-motor-neuron-1-gene-therapy-AveXis; T-BCD541-gene-therapy-AveXis; Telomeric-SMN-gene-therapy-AveXis

Latest Information Update: 17 May 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

  • 21 May 2018 Novartis announces intention to submit regulatory applications in European Union and Japan in 2019
  • 15 May 2018 AveXis has been acquired by Novartis
  • 24 Apr 2018 Efficacy and safety data from the phase I trial in Spinal muscular atrophy released by AveXis
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