Neuren Pharmaceuticals gains FDA orphan drug status for brain injury treatment
Pontos principais
- Neuren Pharmaceuticals secured US FDA Orphan Drug Designation for NNZ-2591 to treat neonatal hypoxic-ischemic encephalopathy.
- Shares in the company rose to $20.74 following the regulatory announcement.
- The company is performing safety studies to submit an Investigational New Drug application in the first half of 2027.
Neuren Pharmaceuticals (ASX:NEU) received a US Food and Drug Administration Orphan Drug Designation for its compound NNZ-2591 to treat neonatal hypoxic-ischemic encephalopathy.
The special status provides seven years of marketing exclusivity and waives prescription drug user fees for the marketing application.
“We now look forward to discussing the development path with the FDA and preparing the IND application for a clinical study in HIE, a devastating condition with serious need,” said Neuren Pharmaceuticals CEO Jon Pilcher.
The company is conducting an animal safety study before submitting an Investigational New Drug application in the first half of 2027.
Following the announcement, the Neuren Pharmaceuticals share price was up at $20.74.
Neonatal hypoxic-ischemic encephalopathy is a brain injury caused by a lack of oxygen or blood flow around birth.
Neuren Pharmaceuticals is also developing NNZ-2591 for Phelan-McDermid, Pitt-Hopkins, and Angelman syndromes.
