The US Food and Drug Administration has granted Prometic Life a Rare Paediatric Disease Designation to its Inter-Alpha-Inhibitor-Proteins (IaIp) for the treatment of Necrotizing Enterocolitis (NEC).
In addition to this latest designation, IAIP has also been granted an Orphan Drug Designation by the FDA.
“This is the second paediatric 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 Pierre Laurin, Prometic President and CEO.
“The combination of paediatric and orphan drug designations provides us with valuable commercial incentives to continue expanding our pipeline of orphan drugs.”
IaIp are endogenous proteins that control excessive inflammatory responses to toxins, infectious organisms, tissue and organ damage. An inverse correlation between IaIp levels in blood plasma and disease severity / mortality has been demonstrated in humans with sepsis.
In a gold-standard animal model proven to emulate NEC in humans, the supplementation of IaIp significantly increased the study subjects’ survival rates.
The FDA grants Rare Paediatric Disease Designations for serious or life-threatening diseases wherein the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.
If and when approved, Prometic’s IaIp replacement therapy could be eligible to receive a rare paediatric disease priority review voucher.