GNT Pharma seeks orphan drug designation for cardiac arrest treatment

GNT Pharma, a local biotech venture, said it has applied to the Ministry of Food and Drug Safety (MFDS) for the orphan drug designation and marketing approval of its cardiac arrest treatment, Gantinel Injection (ingredient: nelonemdaz potassium).

Gantinel is a novel dual-target brain cell protective drug that simultaneously blocks acute glutamate neurotoxicity and delayed reactive oxygen toxicity, two primary causes of brain damage and disability after a stroke.

The MFDS’ orphan drug process is based on clinical trial data demonstrating reasonable predictions of efficacy and safety, with a requirement for phase 3 trial data submission within a set period.

GNT Pharma’s application follows successful results from a phase 2 clinical trial involving 105 cardiac arrest patients undergoing therapeutic hypothermia post-cardiopulmonary resuscitation (CPR), which aimed to verify the safety and efficacy of Gantinel.

Despite hypothermia treatment, cardiac arrest patients typically suffer significant white matter brain damage within five days after resuscitation, often resulting in severe disability or death.

The trial results showed that patients who received Gantinel within four hours of CPR showed a significant reduction in brain white matter damage compared to the placebo group.

Notably, Gantinel markedly reduced damage to critical neural circuits such as the corpus callosum and fornix, which are responsible for interhemispheric communication and memory information transfer.

Further results also showed a dose-dependent decrease in disability and death rates at 90 days among patients administered Gantinel versus those who received a placebo.

Those who received low and high doses experienced a 36.6 percent and 54.8 percent improvement in regaining independence post-treatment. In terms of coma or death, patients treated with low and high doses saw reductions of 31.3 percent and 37.6 percent.

“For the first time, a clinical trial of cardiac arrest patients has shown that a brain cell-protecting drug significantly reduces disability and death by preventing damage to the nerve fibers that carry information to the brain,” GNT Pharma CEO Gwag Byoung-joo said. “As a result, we have applied for orphan drug approval and accelerated review for Gantinel, a brain cell protection drug that is expected to reduce disability and death in cardiac arrest patients who receive hypothermia treatment after CPR.”