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ARIMOCLOMOL CITRATE
Arimoclomol citrate is a marketed drug specifically indicated for the neurological manifestations of Niemann-Pick disease type C (NPC). Its key strength lies in being one of the few treatments available for this rare condition, providing a unique therapeutic option. The primary risk is the expiration of the key composition patent in 2028, which could lead to increased competition from generics.
At a glance
| Generic name | ARIMOCLOMOL CITRATE |
|---|---|
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 2024 |
Approved indications
- Neurological manifestations of NPC
Common side effects
- Upper respiratory tract infection
- Diarrhea
- Decreased weight
Serious adverse events
- Urticaria and angioedema
- Increased serum creatinine
- Hypersensitivity reactions
- Thrombocytopenia
Drug interactions
- OCT2 substrates
Key clinical trials
- Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C (PHASE2, PHASE3)
- Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial (PHASE3)
- Arimoclomol in Amyotropic Lateral Sclerosis (PHASE3)
- Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial (PHASE3)
- Study of Arimoclomol in Inclusion Body Myositis (IBM) (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ARIMOCLOMOL CITRATE CI brief — competitive landscape report
- ARIMOCLOMOL CITRATE updates RSS · CI watch RSS