Tag Archives: HIE

FDA Grants Orphan Status to Experimental Drug for Newborn Brain Injury

The FDA has just granted orphan drug designation* to NNZ-2591 (ercanetide), an experimental drug from Australia’s Neuren Pharmaceuticals, for neonatal hypoxic-ischemic encephalopathy (HIE). NNZ-2591 is an oral solution that, as stated in the Neuren Pharmaceuticals website “is meant to mimic a natural brain molecule that regulates the growth factor IGF-1 and calms inflammation…. NNZ-2591 exert[s] diverse effects which can help to control or normalize abnormal biological processes in the brain. This means that the target is to have a broad impact on the condition rather than aiming to treat one symptom.”

Illustrated side view of the human brain and skull with glowing blue outlines

HIE is a brain injury caused when a baby’s brain is starved of oxygen or blood flow during birth. It is rare, striking about 1 to 1.7 of every 1,000 US births, according to US studies in the American Journal of Obstetrics & Gynecology and Pediatric Neurology. Given its rarity, it is unsurprising, but unfortunate, that no drug is currently approved to treat HIE. The standard treatment protocol is cooling the baby for about 72 hours. Even so, it is reported that 40 – 45 percent of survivors have significant impairment by age two. The hope is that this new medicinal treatment can calm immediate acute injury and prevent lasting damage.

“We now look forward to discussing the development path with the FDA and preparing the IND [Investigational New Drug] application [in the 2027] for a clinical study in HIE, a devastating condition with serious need,” said Neuren CEO Jon Pilcher.

* “The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including: tax credits for qualified clinical trials, exemption from user fees, potential seven years of market exclusivity after approval.” (fda.gov)

** “Because a sponsor will probably want to ship the investigational drug to clinical investigators in many states, it must seek an exemption from that legal requirement. The IND is the means through which the sponsor technically obtains this exemption from the FDA.” (fda.gov)

Birth Brain Injury at Army Hospital Results in $11 Million Settlement

Legal settlement papers for birth brain injury

On September 11, 2021, Sarah Panter delivered a daughter at Blanchfield Army Community Hospital at Fort Campbell, on the border of Kentucky and Tennessee. She was then given Pitocin, a synthetic medication version of oxytocin used to induce pregnancy.  Monitors signaled fetal distress, a noted effect of the drug, and Panter’s attorneys say a C-section should then have been offered, to prevent the infant from injury. Instead, hours later, a midwife delivered the baby, who initially didn’t breathe.

Panther’s daughter now lives with hypoxic-ischemic encephalopathy, brain damage that occurs when a newborn’s brain does not get enough oxygen or blood flow before, during, or right after birth. Additionally, the child lives with cerebral palsy, epilepsy and cortical blindness, known consequences of brain injury. She also cannot walk or sit unaided, uses a feeding tube, and needs round-the-clock nursing for life.

While birth injury is known to “only” be about 2% for normal vaginal delivery and 1% for cesarean delivery, statistics in the United States report that that birth injury occurs in about 25.3 to 31.1 cases per 1000 hospital births. Because the head is typically the first part to enter the birth canal, it is also the most vulnerable and, therefore, common birth injury, as recognized by various studies available in the NLM database.

In September 2026, the Justice Department agreed to an $11 million settlement funding a lifetime-care trust. While the government settled mid-trial, prior to the judge’s ruling, it has not formally accepted responsibility.