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 8Latest Information Update: 04 Nov 2017
Price :
$50
*
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.
- 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