Latest Information Update: 19 Nov 2003
At a glance
- Originator Vertex Pharmaceuticals
- Class Small molecules
- Mechanism of Action Histone deacetylase inhibitors
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
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.
Highest Development Phases
- Discontinued Alpha 1-antitrypsin deficiency; Cystic fibrosis; Huntington's disease; Sickle cell anaemia
Most Recent Events
- 19 Nov 2003 Discontinued - Phase-I for Sickle cell anaemia in USA (unspecified route)
- 19 Nov 2003 Discontinued - Preclinical for Huntington's disease in USA (unspecified route)
- 19 Nov 2003 Discontinued - Preclinical for Cystic fibrosis in USA (unspecified route)