[3] Arimoclomol, in combination with miglustat, is indicated for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC) in adults and children two years of age and older.

[10] Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treat insulin resistance[11][12] and diabetic complications such as retinopathy, neuropathy and nephropathy.

Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) was an experimental drug developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California.

[2] 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.

[14][15] In addition, the FDA has granted priority review, rare pediatric disease, fast track, and breakthrough therapy designations to arimoclomol.