Trehalose granted orphan drug designation in the US for the treatment of amyotrophic lateral sclerosis

Trehalose is a low molecular weight disaccharide that crosses the blood brain barrier, stabilises proteins and activates autophagy (the process that clears material from cells) through activation of Transcription Factor EB. A placebo-controlled Phase III study is planned.

SPS commentary:

Trehalose has previously been granted Orphan Drug Designation from European Medicines Agency and the FDA for Sanfilippo syndrome, spinocerebellar ataxia type 3 and oculopharyngeal muscular dystrophy.

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