Prometic Life Sciences has been granted Rare Pediatric Disease Designation by the FDA for its potential Inter-Alpha-Inhibitor-Proteins (IaIp) therapy for the treatment of necrotizing enterocolitis (NEC).
"This is the second pediatric designation which our plasma-derived therapeutics have received from the FDA, demonstrating the capacity of our plasma purification platform to generate a variety of drug candidates targeting unmet medical needs for children with rare diseases," said Mr. Pierre Laurin, President and Chief Executive Officer of Prometic in a press release
is a condition that damages the intestinal tract of infants, and is more common in premature newborns. The inflammatory bowel condition destroys the wall of the intestine, leading to stool spillage into the infant’s abdomen. This can cause overwhelming infection in the infant causing death.
Symptoms of NEC include abdominal distension, bloody stools, vomiting bile-stained fluid, and gas in the bowel wall identified through x-ray. Newborns with the condition will have temperature instability, lethargy, and early signs of sepsis.
While the cause of the condition is unknown, treatment options vary from ending feedings, passing a small tube into the stomach to relieve gas, and administering intravenous fluids. Approximately 8,000 to 12,000 births in the United States are affected by NEC with mortality rates ranging from 15% to 30%.
NEC also has a high economic cost, and accounts for nearly one-fifth of neonatal expenditures and an annual $5 billion per year in hospitalizations in the United States. The average cost of hospitalization per patient is $73,000 with a length of over 22 days for premature infants. Surgery to correct the intestine wall costs patients an additional $186,000 and requires a stay of over two months.
Prometic Life Sciences has developed IaIp, serine proteases inhibitors that modulate endogenous protease activity to reduce the risk of severe sepsis. The IaIp therapy includes endogenous proteins that control excessive inflammatory responses to toxins, infectious organisms, and tissue and organ damage.
In numerous preclinical sepsis models, IaIp proved to be successful in protecting newborns with NEC and increased survival rates. In addition, IaIp has also received Orphan Drug Designation by the FDA.
"The combination of pediatric and orphan drug designations provides us with valuable commercial incentives to continue expanding our pipeline of orphan drugs,” Laurin concluded. “We look forward to working closely with the FDA to bring this innovative therapy to pediatric patients. The costs associated with this clinical program will not impact our FY2018."