Arimoclomol

Arimoclomol
Clinical data
Trade namesMiplyffa
AHFS/Drugs.comMiplyffa
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
  • N-[(2R,Z)-2-hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride 1-oxide
CAS Number
PubChem CID
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC14H20ClN3O3
Molar mass313.78 g·mol−1
3D model (JSmol)
  • O[C@H](CN1CCCCC1)CO\N=C(\Cl)c2ccc[n+]([O-])c2
  • InChI=1S/C14H20ClN3O3/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17/h4-5,8-9,13,19H,1-3,6-7,10-11H2/b16-14+/t13-/m1/s1 checkY
  • Key:SGEIEGAXKLMUIZ-ZPTIMJQQSA-N checkY
 ☒NcheckY (what is this?)  (verify)

Arimoclomol, sold under the brand name Miplyffa, is a medication for the treatment of Niemann–Pick disease type C.[1][2] It is taken by mouth.[1]

The most common side effects include upper respiratory tract infection, diarrhea, and decreased weight.[2]

Arimoclomol was approved for medical use in the United States in September 2024, when the FDA approved the first treatment for Niemann-Pick Disease, Type C[1][2][3]

  1. ^ a b c d "Miplyffa- arimoclomol citrate capsule". DailyMed. 2 October 2024. Retrieved 5 October 2024.
  2. ^ a b c "FDA Approves First Treatment for Niemann-Pick Disease, Type C". U.S. Food and Drug Administration (FDA) (Press release). 20 September 2024. Retrieved 20 September 2024. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ^ Commissioner Oo (20 September 2024). "FDA Approves First Treatment for Niemann-Pick Disease, Type C". FDA. Retrieved 29 October 2024.