Novel Small Molecule PBT434 Receives Orphan Drug Designation for Treatment of Multiple System Atrophy


The Food and Drug Administration (FDA) has granted orphan drug designation for the molecule PBT434 (Prana Biotechnology LTD,Melbourne, Australia) as a potential treatment for multiple system atrophy (MSA) for which there are no approved therapies. The drug prevents iron-mediated protein aggregation including that of tau and a-synuclein. In animal models of a-synucleinopathies it reduced markers of oxidative stress and a-synuclein aggregation and improved movement. 

The orphan drug designation gives 7 years of exclusivity for PBT434 and marks the first such designation to a drug for treating MSA. 

A Phase 1 clinical trial of PBT434 will be completed this year. “We are pleased that the FDA has acknowledged the importance of PBT434 as a potential treatment for MSA,” said Dr David Stamler, chief medical officer at Prana Biotechnology Ltd. “This recognition … positions us strongly to accelerate the development of PBT434 for this devastating condition.”


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