Drug Profile


Alternative Names: PRXASL

Latest Information Update: 06 Dec 2017

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At a glance

  • Originator PhaseRx
  • Class
  • Mechanism of Action Enzyme replacements
  • Orphan Drug Status

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

    Yes - Inborn urea cycle disorders
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Inborn urea cycle disorders

Most Recent Events

  • 28 Nov 2017 The European Medicines Agency's Committee for Orphan Medicinal Products recommends orphan drug designation for PRX-ASL in European Union for Inborn urea cycle disorders (argininosuccinate lyase deficiency)
  • 20 Sep 2017 PRX ASL receives Orphan Drug status for Inborn urea cycle disorders in USA
  • 08 Nov 2016 Preclinical pharmacodynamics and adverse events data released by PhaseRx
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