Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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

Next Previous
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

Price : $50 *
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

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)
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Legal
Back to top