Research programme: recombinant human acid ceramidase - Plexcera Therapeutics

Drug Profile

Research programme: recombinant human acid ceramidase - Plexcera Therapeutics

Alternative Names: Recombinant human acid ceramidase; rhAC; RVT-801

Latest Information Update: 18 Oct 2017

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At a glance

  • Originator Icahn School of Medicine at Mount Sinai
  • Developer Enzyvant Sciences; Plexcera Therapeutics
  • Class Ceramidases
  • Mechanism of Action Acid ceramidase replacements
  • Orphan Drug Status

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

    Yes - Farber lipogranulomatosis
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Farber lipogranulomatosis
  • Discontinued Cystic fibrosis

Most Recent Events

  • 16 Oct 2017 Recombinant human acid ceramidase receives Orphan Drug status for Farber lipogranulomatosis in European Union before October 2017
  • 16 Oct 2017 Enzyvant plans a clinical trial for Farber lipogranulomatosis in 2018
  • 02 Aug 2016 Discontinued - Preclinical for Cystic fibrosis in USA (Inhalation)
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