arimoclomol
Trade name: Miplyffa
Small moleculeapprovedOrphan Drug FDAFast Track FDABreakthrough Therapy FDA
Approved
Sep 20, 2024
Arimoclomol, sold under the brand name Miplyffa, is a medication used for the treatment of Niemann–Pick disease type C. It is taken by mouth. — Wikipedia
Clinical trial activity
11 trials · 7 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
7
Phase 3
6
Phase 4
0
Earliest trial started Oct 1, 2005 (NCT00244244)
Timeline
2000s
- Jan 1, 2005
Earliest Phase 2 Sponsor(trial)
2010s
- Jan 13, 2015
Orphan Drug Designation
- Jan 1, 2018
Earliest Phase 3 Sponsor(trial)
2020s
- Sep 20, 2024
Zevra Therapeutics — Earliest FDA Approval
- Sep 20, 2024
Zevra Therapeutics — NDA Organization
Indications
Mechanism of action
- Solute carrier family 22 member 2INHIBITOR
SLC22A2 inhibitor
- Unspecified targetOTHER
Unknown
Approval history
- approvedPriority reviewFast trackBreakthroughSep 20, 2024
Chemistry & pharmacology
Loading structure…
SMILES
[O-][n+]1cccc(/C(Cl)=N/OC[C@H](O)CN2CCCCC2)c1- Mol. weight
- 313.79 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
First in class
Sources
- WikipediaArimoclomol ↗
- ChEMBLCHEMBL4298149 ↗
- ChEMBLCHEMBL2107726 ↗
Also known as
- arimoclomol
- arimoclomol
- arimoclomol
- arimoclomol
- arimoclomol
- brx220
- brx-220
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04049097 | Phase 3 | May 20, 2019 | Orphazyme |
| NCT03836716 | Phase 3 | Apr 30, 2019 | Orphazyme, University of Miami |
| NCT03491462 | Phase 3 | Jul 31, 2018 | Orphazyme, University of Miami |
| NCT03746587 | Phase 2 | Jun 6, 2018 | Jaslok Hospital and Research Centre, Mumbai, India, Orphazyme |
| NCT02753530 | Phase 2 | Aug 1, 2017 | University of Kansas |
| NCT02612129 | Phase 2/Phase 3 (Phase 3) | Jun 1, 2016 | Orphazyme |
| NCT00706147 | Phase 2/Phase 3 (Phase 3) | Jan 1, 2009 | Harvard University, University of Miami |
| NCT00769860 | Phase 2/Phase 3 (Phase 3) | Sep 1, 2008 | University of Kansas |
| NCT00561366 | Phase 2 | Nov 16, 2007 | CytRx, Harvard University, State University of New York, Syracuse |
| NCT00244244 | Phase 2 | Oct 1, 2005 | CytRx, Harvard University, State University of New York, Syracuse |
| NCT04316637 | N/A | — | Orphazyme |
Showing 11 of 11 trials
Page 1 / 1
Organizations
Research & Development (7)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Orphazyme | For profit | 6 | 6 | 3 | 2016 |
| Harvard University | Academic/Hospital | 3 | 0 | 2 | 2005 |
| University of Miami | Academic/Hospital | 3 | 1 | 1 | 2009 |
| CytRx | For profit | 2 | 2 | 1 | 2005 |
| State University of New York, Syracuse | Academic/Hospital | 2 | 0 | 1 | 2005 |
| University of Kansas | Academic/Hospital | 2 | 2 | 2 | 2008 |
| Jaslok Hospital and Research Centre, Mumbai, India | Academic/Hospital | 1 | 0 | 1 | 2018 |
7 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Zevra Therapeutics | For profit | NDA | Sep 20, 2024 |