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Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)

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Trial Profile

Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 03 Apr 2025

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At a glance

  • Drugs Apitegromab (Primary)
  • Indications Spinal muscular atrophy
  • Focus Proof of concept; Therapeutic Use
  • Acronyms TOPAZ
  • 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.
  • 08 Jan 2025 According to a Scholar Rock media release, the company plans to submit a U.S. Biologics License Application and a European Union marketing authorization application in quarter one this year if approved, with the company preparing for a U.S. launch expected in 4Q 2025 and a European launch to follow.
  • 07 Oct 2024 According to a Scholar Rock media release, the Company plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025.

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