Research programme: neurodegenerative disorders therapeutics - BioMarin

Drug Profile

Research programme: neurodegenerative disorders therapeutics - BioMarin

Alternative Names: BMN 290; Compound 106; Compound 109; Compound 123; Compound 136; Compound 44; Compound 4b; Compound 526; Compound 531; Compound 9b; RGFA 8

Latest Information Update: 04 Nov 2017

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At a glance

  • Originator The Scripps Research Institute
  • 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for preclinical development in Friedreich's ataxia in USA
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Huntington's-disease in USA
  • 18 Oct 2017 Biomarin plans to file an IND application for BMN 290 for the treatment of Friedreich's ataxia in the second half of 2018
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