Neuren Pharmaceuticals received notice that the European Commission has adopted the European Medicines Agency (EMA) recommendations to grant Orphan designation for NNZ-2591 in Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome. Orphan designation in the EU enables sponsors to benefit from incentives including free protocol assistance, fee reductions and 10 years of market exclusivity plus two additional years if approved for paediatric use. During that exclusivity period, the EMA and the EU Member States shall not accept another marketing authorisation application for a similar medicinal product in the same therapeutic indication. Neuren plans to commence Phase 2 trials in patients with each of Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome in 2021 to confirm the potential of NNZ-2591 to address the urgent unmet need in these three serious childhood disorders which have no approved medicines. In animal models, treatment with NNZ-2591 corrected all deficits in mice with each syndrome and had no effect on normal mice.