Neuren Pharmaceuticals Ltd. said its drug candidate NNZ-2591 was awarded orphan-drug status by the U.S. Food and Drug Administration to treat Angelman syndrome.
The Australian company is developing NNZ-2591 to treat Angelman syndrome, Phelan-McDermid syndrome and Pitt Hopkins syndrome, for which there are no approved therapies.
All three are debilitating childhood disorders. Each disorder is caused by a mutation or deletion in a different gene or chromosomal region, but they share common symptoms and an underlying problem in the connections and signaling between brain cells.
NNZ-2591 aims to restore normal connections and signaling.
Neuren previously announced positive results for NNZ-2591 in separate mouse models for all three genetic conditions.
The company has filed applications seeking orphan-drug designation for the experimental therapy from the FDA for treating Phelan-McDermid syndrome and Pitt Hopkins syndrome.
Clinical trials on NNZ-2591 are expected to begin in 2020.
The FDA grants orphan-drug status to a therapy that treats a rare disease or condition. The special status provides seven years of marketing exclusivity, plus six months if approved for use in children in addition to other incentives.
