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Onasemnogene abeparvovec - AveXis/Nationwide Children's Hospital

Drug Profile

Onasemnogene abeparvovec - AveXis/Nationwide Children's Hospital

Alternative Names: AAV9-CBA-SMN1-gene-therapy-AveXis; Adeno-associated-serotype-9-chicken-beta-actin-survival-motor-neuron-gene-therapy-AveXis; AVXS 101; ChariSMA™; onasemnogene abeparvovec-xioi; 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; ZOLGENSMA

Latest Information Update: 22 Jul 2019

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer 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

  • Marketed Spinal muscular atrophy

Most Recent Events

  • 18 Jul 2019 Launched for Spinal muscular atrophy (In infants, In neonates) in USA (IV) after May 2019
  • 05 Jun 2019 Onasemnogene abeparvovec is approved and is no longer available through the expanded-access programme for Spinal Muscular Atrophy in USA (NCT03955679)
  • 27 May 2019 Onasemnogene abeparvovec-xioi has a boxed warning of acute serious liver injury
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