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

Drug Profile

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: 07 Mar 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

  • 04 Mar 2024 Efficacy and adverse events data from a phase III SMART trial in Spinal muscular atrophy released by Novartis
  • 13 Jun 2023 Novartis Pharmaceuticals completes the phase III SMART trial in Spinal muscular atrophy (In adolescents, In children, In infants) in Australia, Belgium, France, Portugal, United Kingdom, Taiwan, Italy, Germany, Canada and USA (IV) (NCT04851873) (EudraCT2020-005995-37)
  • 22 Apr 2023 Adverse events and efficacy data from a phase III trial in spinal muscular atrophy presented at the 75th Annual Meeting of the American Academy of Neurology 2023 (AAN-2023)
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