Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) has received notice from the European Medicines Agency (EMA) of positive opinions for all three Orphan designation applications that were submitted for NNZ-2591 in Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome.
Under the Orphan procedure timetable, the European Commission is scheduled to issue the decisions in January. 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. Neuren CEO Jon Pilcher commented:
“We are pleased to have received no questions following the EMA review of our compelling pre-clinical results and the rationale for treatment with NNZ2591. We move forward to Phase 2 trials with increased confidence now that we have Orphan designation for all three indications in the two largest markets. We look forward to engaging with both the FDA and the EMA as we strive to develop an effective treatment for patients and families living with these debilitating conditions.”
