An Open-Label Extension Study to Evaluate the LongTerm Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

Trial Profile

An Open-Label Extension Study to Evaluate the LongTerm Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

Completed
Phase of Trial: Phase III

Latest Information Update: 25 Aug 2017

At a glance

  • Drugs Migalastat (Primary)
  • Indications Fabry's disease
  • Focus Adverse reactions; Registrational
  • Acronyms AT1001-041
  • Sponsors Amicus Therapeutics; GlaxoSmithKline
  • Most Recent Events

    • 25 Aug 2017 According to an Amicus Therapeutics media release, data will be presented at the 13th International Congress of Inborn Errors of Metabolism 2017.
    • 11 Jul 2017 According to an Amicus Therapeutics media release, the company is preparing the NDA submission under Subpart H, which provides for accelerated approval. Amicus intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies.
    • 11 Jul 2017 According to an Amicus Therapeutics media release, the company plans to submit a new drug application (NDA) to the U.S. FDA for migalastat for Fabry disease in the fourth quarter of 2017. Based on a series of discussions with and written communication received from the FDA, the Agency has informed Amicus that it may now submit an NDA for migalastat.
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