Research programme: histone deacetylase inhibitors - BioMarin

Drug Profile

Research programme: histone deacetylase inhibitors - BioMarin

Alternative Names: BMN-290; HDAC inhibitors - BioMarin

Latest Information Update: 03 Nov 2017

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At a glance

  • Originator Repligen Corporation
  • Developer BioMarin Pharmaceutical; Repligen Corporation
  • Class Small molecules
  • Mechanism of Action Histone deacetylase inhibitors; Transcription factor stimulants
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Friedreich's ataxia
  • No development reported Huntington's disease
  • Discontinued Cognition disorders; Spinal muscular atrophy

Most Recent Events

  • 26 Oct 2017 Preclinical trials in Friedreich's ataxia in USA (PO)
  • 26 Oct 2017 BioMarin announces intention to submit IND for BMN 290 for Freidriech's ataxia in the second half of 2018
  • 16 Jul 2016 No recent reports of development identified for preclinical development in Friedreich's ataxia in USA (PO)
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