Arimoclomol (MIPLYFFA)
Niemann-Pick disease type C (NPC)
MarketedCommercially Available (US)
Key Facts
Indication
Niemann-Pick disease type C (NPC)
Phase
Marketed
Status
Commercially Available (US)
Company
About Zevra Therapeutics
Zevra Therapeutics is a mission-driven, commercial-stage biopharma company focused on rare diseases. Its strategy centers on acquiring and in-licensing promising candidates, then efficiently advancing them through clinical development and regulatory approval using a data-centric approach. The company has successfully transitioned to a commercial entity with two marketed products (MIPLYFFA® and OLPRUVA®) and maintains a late-stage pipeline with significant catalysts, including a Phase 3 trial for celiprolol in VEDS and a filed MAA for arimoclomol in NPC in Europe.
View full company profileTherapeutic Areas
Other Niemann-Pick disease type C (NPC) Drugs
| Drug | Company | Phase |
|---|---|---|
| Nizubaglustat (RAINBOW/NAVIGATE) | Azafaros | Phase 3 |
| IB1001 (N-acetyl-L-leucine) | IntraBio | Phase 3 |
| Arimoclomol | Zevra Therapeutics | Filed |