Drug Profile
Lucerastat - Idorsia Pharmaceuticals
Alternative Names: ACT 434964; OGT 923Latest Information Update: 05 Nov 2023
Price :
$50
*
At a glance
- Originator Actelion Pharmaceuticals
- Developer Idorsia Pharmaceuticals
- Class Piperidines; Small molecules
- Mechanism of Action Glucosylceramide synthase inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase III Fabry's disease
Most Recent Events
- 13 Dec 2021 Idorsia Pharmaceuticals plans a meeting with health authorities to define the regulatory pathway for lucerastat in Fabry disease in the first half of 2022
- 13 Dec 2021 Efficacy and adverse events data from a phase III MODIFY open-label extension trial in Fabry disease released by Idorsia Pharmaceuticals
- 13 Dec 2021 Efficacy and adverse events data from a phase III MODIFY trial in Fabry disease released by Idorsia Pharmaceuticals