Nizubaglustat (RAINBOW/NAVIGATE)
Niemann-Pick Disease Type C (NPC)
Key Facts
About Azafaros
Azafaros is a private, clinical-stage biopharmaceutical company advancing nizubaglustat, a novel oral small molecule with a dual mechanism of action for lysosomal storage disorders. The company has progressed nizubaglustat into Phase 3 pivotal trials for Niemann-Pick type C disease and GM1/GM2 gangliosidoses, building on positive Phase 2 data. Founded on decades of academic research from Leiden University and Amsterdam UMC, Azafaros is backed by a strong syndicate of European life science investors and led by an experienced industry team.
View full company profileAbout Azafaros
Azafaros is a private, clinical-stage biopharmaceutical company advancing nizubaglustat, a novel oral small molecule with a dual mechanism of action for lysosomal storage disorders. The company has progressed nizubaglustat into Phase 3 pivotal trials for Niemann-Pick type C disease and GM1/GM2 gangliosidoses, building on positive Phase 2 data. Founded on decades of academic research from Leiden University and Amsterdam UMC, Azafaros is backed by a strong syndicate of European life science investors and led by an experienced industry team.
View full company profileTherapeutic Areas
Other Niemann-Pick Disease Type C (NPC) Drugs
| Drug | Company | Phase |
|---|---|---|
| IB1001 (N-acetyl-L-leucine) | IntraBio | Phase 3 |
| Arimoclomol (MIPLYFFA) | Zevra Therapeutics | Marketed |
| Arimoclomol | Zevra Therapeutics | Filed |