Prana Biotechnology (ASX:PBT) has announced the US FDA has granted Orphan Drug designation for its lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA).
The company said it 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 said its application was based on the proposed use of PBT434 as a treatment for MSA, including the medical and scientific rationale.
“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 chief medical officer Dr David Stamler.
Prana is conducting a Phase 1 clinical trial of PBT434 and said it expects it to be completed this year.