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Orphan Designation for Multiple System Atrophy Drug

Treatment Improves Motor Function, Preserves Neurons

Prana Biotechnology Ltd has received orphan drug status for its lead molecule PBT434 for the treatment of Multiple System Atrophy (MSA). It is the first time the FDA has granted orphan designation for this degenerative neurological disorder.

PBT434 prevents the accumulation of alpha-synuclein, protects neurons, and improves motor function in a widely accepted animal model of MSA. Clustered forms of alpha-synuclein are a hallmark of Parkinsonian conditions, including this one. Currently, there are no approved treatments for MSA.  

Prana is conducting a phase 1 clinical trial of PBT434 which it expects to be completed in 2019.

Source: Prana Biotechnology Ltd, January 31, 2019 

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