Research programme: Alpha-L-iduronidase gene therapy - BioMarinAlternative Names: Human alpha-L-iduronidase gene therapy - BioMarin
Latest Information Update: 28 Aug 2013
At a glance
- Originator BioMarin Pharmaceutical
- Class Gene therapies
- Mechanism of Action Gene transference; Iduronidase replacements
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 Mucopolysaccharidosis I