Aceneuramic acid

Drug Profile

Aceneuramic acid

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: 25 Mar 2017

Price : $50

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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase III Nonaka distal myopathy
  • Phase II Thrombocytopenia

Most Recent Events

  • 21 Mar 2017 Nobelpharma plans a phase II/III trial for Nonaka distal myopathy in Japan (UMIN000026354)
  • 11 Nov 2016 Regulatory submission withdrawn for Nonaka distal myopathy in European Union (PO)
  • 11 Aug 2016 Phase-II clinical trials in Thrombocytopenia in Israel (PO) (NCT02845609) before August 2016
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