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An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

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An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 03 Apr 2025

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At a glance

  • Drugs Apitegromab (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Acronyms ONYX
  • Sponsors Scholar Rock

    • Most Recent Events

    • 25 Mar 2025 According to a Scholar Rock media release, the USFDA has accepted its Biologics License Application (BLA) for apitegromab, FDA will review the application under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. Apitegromab's regulatory submissions are based on positive efficacy and safety data from the pivotal Phase 3 SAPPHIRE trial,, as well as supportive data from the Phase 2 TOPAZ trial and long-term extension ONYX trial.
    • 20 Feb 2025 Planned End Date changed from 1 Jan 2027 to 2 May 2029.
    • 07 Oct 2024 According to a Scholar Rock media release, following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study.

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