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Miplyffa (ARIMOCLOMOL)
Miplyffa (generic name: ARIMOCLOMOL) is a Small molecule drug developed by Zevra Denmark. It is currently FDA-approved (first approved 2024) for Niemann-Pick disease type C (NPC).
Miplyffa works by activating heat shock proteins to help protect cells from damage.
Miplyffa is a small molecule modality. It is also known by the synonyms ARIMOCLOMOL and BRX-220 FREE BASE.
At a glance
| Generic name | ARIMOCLOMOL |
|---|---|
| Sponsor | Zevra Denmark |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
| First approval | 2024 |
Mechanism of action
The mechanism(s) by which arimoclomol exerts its clinical effects in patients with NPC is unknown.
Approved indications
- Niemann-Pick disease type C (NPC)
Common side effects
- Upper Respiratory Tract Infection
- Diarrhea
- Decreased Weight
- Urticaria
- Angioedema
- Increased Serum Creatinine
- Hypersensitivity Reactions
- Tremor
- Decreased Appetite
- Headache
- Lower Respiratory Tract Infection
- Seizure
Drug interactions
- OCT2 substrates
- OCT2 substrates
- OCT2 substrates
Key clinical trials
- Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C (PHASE2,PHASE3)
- Early Access Program With Arimoclomol in US Patients With NPC
- 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)
- Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 (PHASE2)
- Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (PHASE2,PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Miplyffa CI brief — competitive landscape report
- Miplyffa updates RSS · CI watch RSS
- Zevra Denmark portfolio CI
Frequently asked questions about Miplyffa
What is Miplyffa?
How does Miplyffa work?
What is Miplyffa used for?
Who makes Miplyffa?
What is the generic name of Miplyffa?
When was Miplyffa approved?
What development phase is Miplyffa in?
What are the side effects of Miplyffa?
Related
- Manufacturer: Zevra Denmark — full pipeline
- Therapeutic area: All drugs in Other
- Indication: Drugs for Niemann-Pick disease type C (NPC)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing