Trial Profile
A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Single and Repeat Doses of Sialic Acid Extended Release (SA-ER) Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Status:
Completed
Phase of Trial:
Phase I
Latest Information Update: 06 Nov 2021
Price :
$35
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At a glance
- Drugs Aceneuramic acid (Primary)
- Indications Nonaka distal myopathy
- Focus Adverse reactions
- Sponsors Ultragenyx Pharmaceutical
- 02 Oct 2015 According to Ultragenyx media release, the company has filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for aceneuramic acid prolonged release (AceER; UX001) tablets intended for patients with GNE Myopathy. The submission has been accepted for review and the company expects a decision from the European Commission in the second half of 2016.
- 04 Dec 2014 New trial record