Latest Information Update: 31 Oct 2003
At a glance
- Originator Transkaryotic Therapies
- Mechanism of Action Undefined mechanism
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 Metabolic disorders
Most Recent Events
- 15 Mar 2016 Biomarkers information updated
- 31 Oct 2003 Discontinued - Phase-I for Metabolic disorders in USA (unspecified route)
- 31 Dec 2001 Phase-I clinical trials in Metabolic disorders in USA (unspecified route)