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Science & Pipeline
We are dedicated to developing transformational, patient-focused therapies for rare diseases with limited or no treatment options available, or treatment areas with significant unmet needs. Our expertise and science-driven approach has produced a late-stage rare disease clinical pipeline and multiple commercial products.
NIEMANN-PICK DISEASE TYPE C PROGRAM
| PRODUCT CANDIDATE | INDICATION |  |
MILESTONES |
|---|---|---|---|
| Arimoclomol | Niemann-Pick disease type C (NPC) |
|
Filed Marketing Authorization Application with the European Medicines Agency. |
Vascular Ehlers-Danlos Syndrome Program
| PRODUCT CANDIDATE | INDICATION | |
MILESTONES |
|---|---|---|---|
| Celiprolol New chemical entity for the treatment of COL3A1-positive VEDS. | Vascular Ehlers-Danlos Syndrome (VEDS) |
|
Phase 3 trial ongoing |
Rare Sleep Disorders Program
| PRODUCT CANDIDATE | INDICATION | |
MILESTONES |
|---|---|---|---|
| KP1077 Prodrug candidate for IH and narcolepsy type I & II | Idiopathic Hypersomnia (IH) Narcolepsy |
|
Phase 3 trial ready Phase 3 trial potential |
