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

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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™; Itvisma; 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: 01 Dec 2025

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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

  • 24 Nov 2025 Registered for Spinal muscular atrophy (In adolescents, In children, In the elderly, In adults) in USA (Intrathecal)
  • 24 Nov 2025 Novartis plans to launch Onasemnogene abeparvovec for Spinal muscular atrophy (In adolescents, In children, In adults, In elderly) in USA (Intrathecal)
  • 29 Apr 2025 Novartis Pharmaceuticals completed the phase III STEER trial in Spinal muscular atrophy (In children, In adolescents) in the US, Brazil, China, Colombia, Egypt, Denmark, Greece, India, Italy, Malaysia, Mexico, Russia, Saudi Arabia, Singapore, South Africa, Taiwan, Thailand, United Arab Emirates and Vietnam (Intrathecal) (NCT05089656) (EudraCT2021-003474-31)

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