New Drug Approvals


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Exact mass313.1193
Mol weight313.7799


289893-26-1 (Arimoclomol maleate);

INN 8300

N-[(2R)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride

BRX 220

Arimoclomol maleate is in a phase III clinical trials by Orphazyme for the treatment of Niemann-Pick disease type C (NP-C). It is also in phase II clinical studies for the treatment of amyotrophic lateral sclerosis (ALS).

Arimoclomol(INN; originally codenamedBRX-345, which is a citrate salt formulation ofBRX-220) is an experimental drug developed byCytRx Corporation, a biopharmaceutical company based inLos Angeles, California. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS.[1]TheEuropean Medicines Agency(EMA) andU.S. Food & Drug Administration(FDA) granted orphan drug designation to arimoclomol as a potential treatment forNiemann-Picktype C in 2014 and 2015 respectively.[2][3]

Fig. 1Structures of (±)-bimoclomol (1) and (R)-(+)-arimoclomol…

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