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Aceneuramic acid - Nobelpharma/Ultragenyx Pharmaceutical

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Drug Profile

Aceneuramic acid - Nobelpharma/Ultragenyx Pharmaceutical

Alternative Names: Ace-ER - Ultragenyx; Aceneuramic acid extended-release - Ultragenyx; Acenobel; 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: 02 Apr 2024

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At a glance

  • Originator Nobelpharma
  • Developer Hadassah Medical Organization; Nobelpharma; 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

  • Registered Nonaka distal myopathy
  • No development reported Thrombocytopenia

Most Recent Events

  • 26 Mar 2024 Registered for Nonaka distal myopathy in Japan (PO)
  • 25 Mar 2024 Preregistration for Nonaka distal myopathy in Japan (PO), prior to March 2024
  • 14 Oct 2022 Nobelpharma anticipates regulatory approval for Nonaka-distal-myopathy in March 2024 (Nobelpharma pipeline, July 2023)
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