The Food and Drug Administration has granted Orphan Drug designation to PBT434 (Prana Biotechnology), an investigational agent for the treatment of multiple system atrophy (MSA).
Currently, there are no approved therapies for the treatment of MSA, a rare progressive neurodegenerative disease. Patients with MSA may present with Parkinsonian symptoms, however treatments used in Parkinson’s disease have not been shown to be effective.
In an animal model, PBT434 demonstrated an ability to prevent α-synuclein accumulation, preserve neurons, and improve motor function. A Phase 1 clinical trial is currently underway; Prana expects the study to be completed sometime in 2019.