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: 16 Nov 2017

Price : *
* Final gross price and currency may vary according to local VAT and billing address.

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

  • 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
  • 27 Jul 2017 Ultragenyx announces intention to submit MAA to EMA for Nonaka distal myopathy
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top