Aceneuramic acid - Nobelpharma/ Ultragenyx Pharmaceutical

Drug Profile

Aceneuramic acid - Nobelpharma/ Ultragenyx Pharmaceutical

Alternative Names: Ace-ER - Ultragenyx; Aceneuramic acid extended-release - Ultragenyx; Kepnetic; N-acetylneuraminic acid (anhydride) - Nobelpharma; NANA; Neu5Ac; NPC 09; SA-ER - Ultragenyx; Sialic acid; Sialic acid ER - Ultragenyx; Sialic acid extended release - Ultragenyx; UX 001 Sialic acid; UX-001; UX-001 SA-ER

Latest Information Update: 24 Mar 2018

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At a glance

  • Originator Nobelpharma
  • Developer Hadassah Medical Organization; Japan Agency for Medical Research and Development; Nobelpharma; Tohoku University; Ultragenyx Pharmaceutical
  • Class Neuraminic acids; Sialic acids; Small molecules
  • Mechanism of Action Muscle protein stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Nonaka distal myopathy
  • New Molecular Entity No

Highest Development Phases

  • Phase III Nonaka distal myopathy
  • Phase II Thrombocytopenia

Most Recent Events

  • 30 Sep 2017 Nobelpharma completes a phase II/III trial in Nonaka distal myopathy in Japan (PO) (UMIN000020683)
  • 22 Aug 2017 Top-line adverse events data from a phase III trial in Nonaka distal myopathy released by Ultragenyx Pharmaceutical
  • 22 Aug 2017 Ultragenyx Pharmaceutical plans to discontinue the development of aceneuramic acid for Nonaka distal myopathy
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