Research programme: recombinant human acid ceramidase - Plexcera TherapeuticsAlternative Names: Recombinant human acid ceramidase; rhAC; RVT-801
Latest Information Update: 03 Nov 2016
At a glance
- Originator Icahn School of Medicine at Mount Sinai
- Developer Enzyvant Sciences; Plexcera Therapeutics
- Class Ceramidases
- Mechanism of Action Acid ceramidase 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.
- New Molecular Entity Yes
Highest Development Phases
- Preclinical Farber lipogranulomatosis
- Discontinued Cystic fibrosis