Mucopolysaccharidosis IVIIB therapeutic - Dorphan
Latest Information Update: 15 Jul 2016
At a glance
- Originator DORPHAN S.A.
- Class Imino sugars; Pharmacological chaperones
- Mechanism of Action Glycoside hydrolase 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.
- New Molecular Entity Yes
- Available For Licensing Yes
Highest Development Phases
- Preclinical Mucopolysaccharidosis IV
Most Recent Events
- 15 Jul 2016 Preclinical trials in Mucopolysaccharidosis IV in Switzerland (unspecified route)
- 15 Jul 2016 Mucopolysaccharidosis IVIIB therapeutic - Dorphan is available for licensing as of 15 Jul 2016. http://www.dorphan.com/