Neuren Pharmaceuticals Ltd (ASX:NEU) (OTCMKTS:NURPF) has received European Commission’s (EC) approval granting Orphan designation for its second drug candidate NNZ-2591 used in treating Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome.
Neuren is advancing the development of its NNZ-2591 for these syndromes and has now received orphan drug designation in the United States and the European Union (EU).
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.
Proposed clinical trials
Neuren is conducting a phase 1 clinical trial of NNZ-2591 and plans to initiate Phase 2 trials.
The aim of Phase 2 trials in patients with each of Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome is 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 studies, treatment with NNZ-2591 corrected all deficits in mice with each syndrome and did not affect normal mice.
The company is developing two new drug therapies to treat five serious neurological disorders that emerge in early childhood.