Arimoclomol
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed Niemann-Pick disease type C, adult neurologic onset 4 milestones | |||||
| Refused (EMA) | EU (EMA) | — | Niemann-Pick disease type C, adult neurologic onset | 2026-07-24 | “has adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease type C (NPC)” zevra.com ↗ |
| Filed | EU (EMA) | MIPLYFFA | Niemann-Pick disease type C, adult neurologic onset | 2025-07-28 | “submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C” zevra.com ↗ “Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C” zevra.com ↗ “A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC is under review by the European Medicines Agency (EMA).” zevra.com ↗ “supporting the arimoclomol MAA submission” zevra.com ↗ |
| Rejected (CRL) | US (FDA) | — | Niemann-Pick disease type C, adult neurologic onset | 2021-06-17 | “On June 17, 2021 , Orphazyme received a Complete Response Letter (CRL) from the FDA regarding its NDA for arimoclomol for the treatment of NPC.” zevra.com ↗ |
| Filed | US (FDA) | — | Niemann-Pick disease type C, adult neurologic onset | — | “the arimoclomol NDA which was resubmitted to the FDA in December 2023” zevra.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | MIPLYFFA | — | 2024-09-20 | fda.gov ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 24 trials · 1 missed | ||||||
| Phase 2 | NCT03746587 | Jun 2018 → Apr 2020 | Gaucher disease type I, Gaucher disease type III | ZevraDenmark | Terminated | No outcome recorded |
| Phase 2 | NCT02753530 | Aug 2017 → Jan 2021 | inclusion body myositis | ZevraDenmark | Completed | Missed primary |
| Phase 2 | NCT00244244 | Oct 2005 → Jan 2007 | amyotrophic lateral sclerosis | CytRx | Completed | No outcome recorded |
| Phase 2 | NCT00561366 | — | amyotrophic lateral sclerosis | CytRx | Withdrawn | No outcome recorded |
| Phase 2/31 trial · 1 met primary | ||||||
| Phase 2/3 | NCT02612129 | Jun 2016 → Jun 2018 | Niemann-Pick disease type C | ZevraDenmark | Completed | Met primary |
| Phase 33 trials · 1 missed | ||||||
| Phase 3 | NCT03836716 | Sep 2019 → Jul 2021 | amyotrophic lateral sclerosis | ZevraDenmark | Terminated | No outcome recorded |
| Phase 3 | NCT04049097 | May 2019 → Nov 2021 | inclusion body myositis | ZevraDenmark | Terminated | No outcome recorded |
| Phase 3 | NCT03491462 | Jul 2018 → Dec 2020 | amyotrophic lateral sclerosis | ZevraDenmark | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT04316637 | — | Niemann-Pick disease type C | ZevraDenmark | Available | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 2/3 | NCT00706147 Comparator | Jan 2009 → Dec 2014 | familial amyotrophic lateral sclerosis | University of Miami | Completed | No outcome recorded |
| Phase 2/3 | NCT00769860 Comparator | Sep 2008 → Sep 2012 | inclusion body myositis | Richard Barohn, MD | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 21
All press releases naming this drug 29 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Arimoclomol | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03491462 ↗ |
| Known as | Arimoclomol (BRX-345) | “Arimoclomol (BRX-345)” NCT00706147 ↗ |
| Known as | ARIMOCLOMOL CITRATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL2107726 ↗ |
| Known as | BRX-220 FREE BASE | ChEMBL registry synonym — accepted as the source's own label CHEMBL2107726 ↗ |
| Known as | Miplyffa | ChEMBL registry synonym — accepted as the source's own label CHEMBL2107726 ↗ |
| Known as | Orphazyme a/s | ChEMBL registry synonym — accepted as the source's own label CHEMBL2107726 ↗ |
| Action | Activate | “upregulates "molecular chaperones"” NCT00561366 ↗ |
| Modality | Small molecule | “Arimoclomol is a small molecule that upregulates "molecular chaperones" in cells under stress.” NCT00244244 ↗ |
| Route | Oral | “Participants received arimoclomol capsules orally three times a day (TID) for 12 months.” NCT02612129 ↗ |