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| Trade names | Miplyffa |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | By mouth |
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| Chemical and physical data | |
| Formula | C14H20ClN3O3 |
| Molar mass | 313.78 g·mol−1 |
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Arimoclomol, sold under the brand nameMiplyffa, is amedication used for the treatment ofNiemann–Pick disease type C.[1][2] It is takenby mouth.[1]
The most common side effects includeupper respiratory tract infection,diarrhea, anddecreased weight.[2]
Arimoclomol was approved for medical use in the United States in September 2024.[1][2] The USFood and Drug Administration (FDA) considers it to be afirst-in-class medication.[3]
Arimoclomol, in combination with miglustat, isindicated for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC) in adults and children two years of age and older.[1][2]
The most common side effects of arimoclomol include upper respiratory tract infection, diarrhea, and decreased weight.[2]
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation ofmolecular chaperones.[medical citation needed] Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.[medical citation needed]
Arimoclomol activates theheat shock response.[4][5][6][7][8][9] It is believed to act atHsp70.[10]
Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treatinsulin resistance[11][12] and diabetic complications such asretinopathy,neuropathy andnephropathy. Later, the compound, along with other small molecules, was screened for further development by Hungarian firm Biorex, which was sold to CytRx Corporation, who developed it toward a different direction from 2003.
Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) was an experimental drug developed byCytRx Corporation, a biopharmaceutical company based in Los Angeles, California.[2] In 2011, the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS.[13]
In September 2024, the USFood and Drug Administration (FDA) granted approval of arimoclomol to Zevra Therapeutics.[2]
The FDA approved arimoclomol based on results of a randomized, double-blind, placebo-controlled 12-month trial in participants 2 to 19 years of age who had a molecularly confirmed diagnosis of Niemann-Pick disease, type C. Fifty participants were randomized 2:1 to treatment with weight-adjusted arimoclomol (31 to 124 mg) or placebo orally three times per day.[14] Among these 50 participants, 39 (78%) received miglustat as background treatment in the trial.[2] The trial was conducted at 14 sites in Europe, Great Britain, and the United States.[14]
Efficacy was demonstrated by the rescored 4-domain Niemann-Pick disease, type C Clinical Severity Scale (R4DNPCCSS) score in the participants who used miglustat as their background treatment. The R4DNPCCSS is a measure of Niemann-Pick disease, type C disease progression that looks at four items that participants with Niemann-Pick disease, type C, their caregivers and physicians have identified as most relevant including ambulation, speech, swallow and fine motor skills. Higher scores signify a greater severity of the disease. Compared to placebo, arimoclomol resulted in a slower disease progression as measured by the R4DNPCCSS score.[2]
TheEuropean Medicines Agency (EMA) and USFood & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment forNiemann-Pick type C in 2014 and 2015 respectively.[15][16] In addition, the FDA has granted priority review, rare pediatric disease, fast track, and breakthrough therapy designations to arimoclomol.[2]
Arimoclomol was approved for medical use in the United States in September 2024.[2][17]
Arimoclomol is theinternational nonproprietary name (INN).[18]
Arimoclomol has been shown to extend life in an animal model of ALS[19] and was well tolerated in healthy human volunteers in aPhase I study. CytRx is currently conducting a Phase II clinical trial.[20]
Arimoclomol also has been shown to be an effective treatment in an animal model of Spinal Bulbar Muscular Atrophy (SBMA, also known as Kennedy's Disease).[21]
This article incorporates text from this source, which is in thepublic domain. This article incorporates text from this source, which is in thepublic domain.
