Prana Biotechnology Ltd (ASX: PBT, NASDAQ: PRAN) has today announced the US Food and Drug Administration (FDA) has granted Orphan Drug designation for its lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA). This is the first time the FDA has granted orphan designation to a drug for the treatment of MSA.
Orphan Drug designation by the FDA entitles Prana to seven years of market exclusivity for the use of PBT434 in the treatment of MSA and qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing.
Prana’s successful application was based on the proposed use of PBT434 as a treatment for MSA, including the medical and scientific rationale. The application articulated how PBT434 prevents α-synuclein accumulation, preserves neurons, and improves motor function in a widely accepted animal model of MSA. Alpha-synuclein is of great interest because aggregated forms of the protein are a pathological hallmark of Parkinsonian conditions, including MSA, and it represents a recognised therapeutic target by the scientific community.
“We are pleased that the FDA has acknowledged the importance of PBT434 as a potential treatment for MSA. This recognition, in conjunction with the recent investment from Life Biosciences, positions us strongly to accelerate the development of PBT434 for this devastating condition,” said Dr David Stamler, Chief Medical Officer.
Prana has identified a clear unmet medical need with no approved treatments specifically for MSA. Prana is conducting a Phase 1 clinical trial of PBT434 and expects it to be completed this year.