Neuren Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration for NNZ-2591 as a treatment for neonatal hypoxic-ischemic encephalopathy, or HIE, a brain injury that affects newborns.
The FDA can grant orphan drug designation to a medicine for a rare disease or condition that affects fewer than 200,000 patients in the United States. The status comes with several incentives. These include seven years of marketing exclusivity and a waiver of the prescription drug user fee when the sponsor files a marketing application.
HIE happens when a baby's brain does not get enough oxygen or blood flow before birth or shortly after it. Many thousands of children are affected each year, and the condition is one of the leading causes of neonatal death and neurodevelopmental disability worldwide. Children who survive can have developmental delays, cognitive impairment, cerebral palsy and seizures. Some develop serious complications that last well into adulthood.
The company is running a juvenile animal safety study, which the FDA requires before an HIE clinical study can begin. Neuren plans to submit an Investigational New Drug application in the first half of 2027.
Neuren chief executive Jon Pilcher said the designation was a significant step for the company. "We are very pleased to receive the important commercial milestone of orphan drug designation. 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."
NNZ-2591 is being developed for several conditions. Besides HIE, it is also being developed for Phelan-McDermid syndrome, Pitt-Hopkins syndrome, and Angelman syndrome, each of which has orphan drug designation.