Tag Archives: rare

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)

When Bears Attack

Rocky forest trail leading toward snow-capped mountains and winding river

On Sept. 14, a 20-year-old bowhunter near Augusta, Montana, surprised a grizzly near a cow carcass. Badly hurt, the injured man still drove himself home. In May, a mother grizzly with cubs mauled two brothers at Yellowstone. The older brother needed facial reconstruction and care for a head wound.

Unfortunately, head injuries are a relatively common consequence of a bear attack, as bears typically strike the head, face or neck first. These strikes can then result in skull fractures and traumatic brain injury. In one study of 417 bear-attack victims, 55% had head injuries. While bear attacks are rare, roughly 11 brown-bear attacks a year occur in North America. These attacks are clustered, with Alaska, Montana, Wyoming and British Columbia known to be grizzly bear backcountry.

Western governors want control of grizzly management to return to the states. “The endangered species list is not a dean’s list,” Wyoming Gov. Mark Gordon said in July. “It’s time that [the grizzly] bear graduates.” Peak season for bear attacks is from July through September, as bears eat up to 20,000 calories a day to fatten up before hibernation.  According to 2024 information found in the NLM database, “upper‐body injury results from bears often attacking from a standing position, targeting the head and neck,” so be wary of a standing bear.

Year round, caution is recommended for those who traverse bear-friendly terrain: carry bear spray, travel in groups and make noise. The U.S. National Park Service and other wildlife authorities advise that if a grizzly attacks, lie face-down with your hands clasped over your neck. Fight back against black bears.