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A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Trial Profile

A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 27 May 2019

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At a glance

  • Drugs Aceneuramic acid (Primary)
  • Indications Nonaka distal myopathy
  • Focus Pharmacodynamics; Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 11 Nov 2016 According to an Ultragenyx Pharmaceutical media release, the company has withdrawn its conditional MAA from the European Medicines Agency for Aceneuramic Acid Prolonged Release for the treatment of adult patients with GNE Myopathy, as the CHMP indicated that the Phase 2 study was encouraging but did not provide a sufficient amount of evidence to support an approval at this time. The company now plans to submit the MAA based on the data from a phase III trial.
    • 27 Jul 2016 According to Ultragenyx media release, the Committee for Medicinal Products for Human Use (CHMP) opinion is expected in the second half of 2016, with a final decision expected in the first half of 2017.
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