Newborn brain injury drug reaches FDA milestone

Neuren Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for a potential treatment for newborn brain injury.

The designation covers NNZ-2591, a drug being developed to treat neonatal hypoxic-ischaemic encephalopathy (HIE), a condition caused by insufficient oxygen or blood flow to a baby’s brain before birth or shortly afterwards.

HIE is one of the leading causes of neonatal death and neurodevelopmental disability worldwide, affecting many thousands of children each year.

Children who survive can experience developmental delays, cognitive impairment, cerebral palsy and seizures, with some facing serious complications that continue well into adulthood.

The FDA can grant orphan drug designation to medicines being developed for rare diseases or conditions affecting fewer than 200,000 patients in the US.

The status provides incentives to support drug development, including seven years of marketing exclusivity and a waiver of the prescription drug user fee when a marketing application is submitted.

The designation does not mean NNZ-2591 has been approved for clinical use.

Neuren is currently conducting a juvenile animal safety study, which the FDA requires before a clinical study involving patients with HIE can begin.

The company plans to submit an Investigational New Drug (IND) application in the first half of 2027.

Jon Pilcher, chief executive of Neuren Pharmaceuticals, said the designation was a significant step for the company.

He said: “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 also being developed for three other conditions: Phelan-McDermid syndrome, Pitt-Hopkins syndrome and Angelman syndrome.

All three indications have already received orphan drug designation.

Clinical testing of NNZ-2591 for HIE has yet to begin, with the company completing the required animal safety study before progressing to the next stage of development.