Corestem said Monday that it has applied for a pre-investigational new drug (Pre-IND) application consultation with the U.S. Food and Drug Administration regarding Neuronata-R Injection, the company's treatment for Lou Gehrig's disease.
|Corestem’s Lou Gehrig disease treatment NeuroNata-R|
Corestem received an orphan drug designation (ODD) for the treatment from the FDA in August 2018.
FDA's ODD is a system that helps smooth the development and approval of therapeutic drugs for rare or life-threatening diseases. ODD drugs receive tax holidays, exemption of license application fees, and seven-year monopoly after obtaining authorization.
The company will meet with FDA officials in July to do the consultation.
NeuroNata-R is a stem cell treatment for Lou Gehrig's disease, which slows down body function's decline by 50 percent after six months of prescription in a clinical trial. The drug is administered twice every four weeks and costs about 60 million won ($55,136) annually.
Although the drug received approval in Korea in 2014, the state-run health insurance review agency has recently refused to grant insurance benefit to NeuroNata-R Injection due to its low cost-effectiveness. The drug reimbursement assessment committee said the treatment's cost-effectiveness was unclear, ruling that it cannot be reimbursed.
"To date, more than 250 patients have been treated with the drug," the company said. "About 55 of them are patients who have come from abroad to receive the treatment."