Miplyffa Orphazyme A/S · Application withdrawn · ATC N07
Therapeutic indication (EMA)
Treatment of Niemann-Pick disease type C (NPC).
Active substance
Arimoclomol
- US FDA 1
- EMA 2
US FDA United States — FDA approvals (Drugs@FDA)
| Product | Strength | Form · route | Applicant | Status | First approved |
|---|---|---|---|---|---|
| MIPLYFFA | EQ 47MG BASE; EQ 62MG BASE; EQ 93MG BASE; EQ 124MG BASE | capsule · oral | ZEVRA DENMARK | Prescription | 20 Sep 2024 |
EMA European Union — centrally authorised medicines
Meplyffa Zevra Denmark A/S · Opinion · ATC N07XX17
Therapeutic indication (EMA)
Treatment of Niemann-Pick disease type C (NPC) in patients aged 6 months and older in combination with miglustat.
Values as published by each source: SFDA human drug list (4-2026, as published by the Council of Health Insurance; indications from the CHI Daman Drug Formulary), Drugs@FDA, EMA medicines data, Health Canada Drug Product Database (Open Government Licence – Canada), CMS NADAC, NHS Drug Tariff Part VIIIA (NHSBSA Copyright, Open Government Licence v3.0), WHO Model List of Essential Medicines (CC BY 3.0 IGO; regrouped by active substance, not created by WHO), EMA PRAC, US FDA safety-related labeling changes and MHRA Drug Safety Update (Open Government Licence v3.0). Sources and reuse. Not a substitute for the official registers or the product information.