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Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

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Trial Profile

Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

Status: Completed
Phase of Trial: Clinical Phase Unknown

Latest Information Update: 27 May 2025

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At a glance

  • Drugs Arimoclomol (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Expanded access; Therapeutic Use
  • Sponsors Orphazyme; Zevra Therapeutics

Most Recent Events

  • 13 May 2025 According to a Zevra Therapeutics Media release, all active patients in the U.S. Expanded Access Program (EAP) for arimoclomol, have been enrolled to receive MIPLYFFA. The EAP has since closed, including locking the database and deactivating study sites.
  • 13 May 2025 Status changed from recruiting to completed.
  • 21 Apr 2025 According to a Zevra Therapeutics Media release, MIPLYFFA became available to patients within 8 weeks after USFDA approval, and with the support of the NPC community the company ensured a smooth transition for all U.S. Expanded Access Program (EAP) participants to MIPLYFFA as a commercial product.

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