AMT 060

Drug Profile

AMT 060

Alternative Names: AAV5-hFIX; AMT-060

Latest Information Update: 17 Jan 2017

Price : $50

At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer St. Jude Childrens Research Hospital; uniQure; University College London
  • Class Gene therapies
  • Mechanism of Action Factor IX stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Haemophilia B
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Haemophilia B

Most Recent Events

  • 09 Jan 2017 uniQure plans a pivotal phase III trial in Haemophilia B in 2018
  • 03 Dec 2016 Adverse events and efficacy data from a phase I/II trial in presented at the 58th American Society of Hematology Annual Meeting (ASH-2016)
  • 27 Jul 2016 Updated efficacy and adverse event data from the low-dose cohort of the phase I/II trial in Haemophilia B released by uniQure
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