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Neuren's therapy wins FDA orphan-drug status to treat rare genetic disorder

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.