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Onasemnogene abeparvovec - Novartis Gene Therapies/Nationwide Children's Hospital

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Onasemnogene abeparvovec - Novartis Gene Therapies/Nationwide Children's Hospital

Alternative Names: AAV9-CBA-SMN1-gene-therapy-Novartis Gene Therapies; Adeno-associated-serotype-9-chicken-beta-actin-survival-motor-neuron-gene-therapy-Novartis Gene Therapies; AVXS 101; ChariSMA™; OAV-101; onasemno-gene abepar-vovec; onasemnogene abeparvovec-xioi; scAAV9.CB.SMN; SMA1-gene-therapy-Novartis Gene Therapies; SMN1-gene-therapy-Novartis Gene Therapies; SMNT-gene-therapy-Novartis Gene Therapies; Spinal-muscular-atrophy-gene-therapy-Novartis Gene Therapies; Survival-motor-neuron-1-gene-therapy-Novartis Gene Therapies; T-BCD541-gene-therapy-Novartis Gene Therapies; Telomeric-SMN-gene-therapy-Novartis Gene Therapies; ZOLGENSMA

Latest Information Update: 31 Dec 2024

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer AveXis; Novartis Gene Therapies
  • Class Gene therapies; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 1 protein replacements
  • 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

  • 30 Dec 2024 Efficacy and adverse event data from the phase III STEER trial in Spinal muscular atrophy released by Novartis
  • 29 Nov 2024 Novartis completes a phase III STRENGTH trial for Spinal muscular atrophy (In Children, Treatment experienced) in Canada, Japan, Netherlands, Australia, Belgium, France, Italy, Spain and the USA (Intrathecal) (NCT05386680)
  • 04 Mar 2024 Efficacy and adverse events data from a phase III SMART trial in Spinal muscular atrophy released by Novartis

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