arimoclomol

Arimoclomol is an orally administered small molecule used in combination with miglustat to treat neurological symptoms in adults and children (2 years and older) with Niemann-Pick disease type C (NPC), a rare inherited lysosomal storage disorder. It acts as a heat shock protein modulator, specifically amplifying the heat shock response (HSR) and increasing production of molecular chaperones such as Heat shock protein 70 (HSP70). These proteins help prevent misfolding and aggregation of other proteins, stabilize lysosomal membranes, enhance sphingolipid-degrading enzyme activity, and protect against cell death under cellular stress conditions. Arimoclomol also activates transcription factors TFEB and TFE3, upregulating CLEAR genes essential for lysosomal function—including NPC1—thereby improving cholesterol trafficking within cells. The drug crosses the blood-brain barrier and has demonstrated neuroprotective effects in animal models. It was developed by Zevra Therapeutics.

Brand names

Miplyffa

Other names

arimoclomol citrate