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Onasemnogene Abeparvovec: First Global Approval.

Sheridan M Hoy1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

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|July 5, 2019
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Summary

Onasemnogene abeparvovec is the first gene therapy approved in the USA for spinal muscular atrophy (SMA). This treatment delivers a functional SMN gene to motor neurons in young children with SMA.

Area of Science:

  • * Gene therapy
  • * Neurology
  • * Pediatrics

Background:

  • * Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder.
  • * SMA is caused by mutations in the SMN1 gene, leading to motor neuron degeneration.
  • * Onasemnogene abeparvovec offers a novel therapeutic approach for SMA.

Purpose of the Study:

  • * To summarize the development milestones of onasemnogene abeparvovec.
  • * To highlight its approval as the first gene therapy for SMA in the USA.
  • * To detail its application in pediatric patients with SMA.

Main Methods:

  • * Development of an adeno-associated viral vector-based gene therapy.
  • * Delivery of a functional human SMN gene to motor neurons.
  • * Intravenous administration of a single dose.

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Main Results:

  • * Onasemnogene abeparvovec received US FDA approval in May 2019.
  • * It is indicated for pediatric patients under 2 years with SMA and bi-allelic SMN1 mutations.
  • * Regulatory assessments are ongoing in the EU and Japan.

Conclusions:

  • * Onasemnogene abeparvovec represents a significant advancement in SMA treatment.
  • * It is the first gene therapy approved for SMA in the United States.
  • * Further development includes an intrathecal formulation.