Australian biotech company Prana Biotechnology has secured Orphan Drug designation for PBT434, its lead molecule, for the treatment of Multiple System Atrophy (MSA).
Prana Biotechnology claims that its application was based on the proposed use of the molecule as a treatment for MSA, including the medical and scientific rationale.
The Australian biotech company said that its application articulated how PBT434 blocks α-synuclein accumulation, preserves neurons, and boosts motor function in a largely accepted animal model of MSA.
According to Prana Biotechnology, alpha-synuclein is of great interest as aggregated types of the protein are a pathological hallmark of MSA and other Parkinsonian conditions. Alpha-synuclein is also a recognized therapeutic target by the scientific community, said the Australian biotech company.
Prana Biotechnology claims that it has identified a clear unmet medical need which has no approved treatments yet particularly for MSA. The Aussie biotech company is holding a phase 1 clinical trial of PBT434 which it expects to wrap up this year.
Dr David Stamler – Chief Medical Officer of Prana Biotechnology said: “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.”
The orphan drug designation from FDA gives Prana Biotechnology seven years of market exclusivity for PBT434 to be used in the treatment of MSA. It also qualifies the sponsor of the drug for multiple development incentives of the Orphan Drug Act, which includes tax credits for qualified clinical testing.
Last month, the Australian biotech company raised about A$44.5m ($31.4m) in an investment round led by Boston-based Life Biosciences.