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Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

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Trial Profile

Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 16 Sep 2022

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; First in man; Registrational
  • Acronyms START
  • Sponsors Novartis Gene Therapies

Most Recent Events

  • 13 Mar 2022 Results of post-hoc analysis from START, STR1VE-EU and STR1VE-US studies were presented at Novartis media release.
  • 19 Nov 2020 Results of matching adjusted indirect comparison of Onasemnogene Abeparvovec and Nusinersen presented at the 23rd Annual European Congress of the International Society for Pharmacoeconomics and Outcomes Research
  • 19 May 2020 According to an AveXis media release, based on the STR1VE-US and START trials, the European Commission (EC) granted conditional approval for Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

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