Prometic announces that it has received orphan drug designation status for Inter-Alpha-Inhibitor-Proteins (IaIp) in the treatment of necrotizing enterocolitis by the US Food and Drug Administration (FDA).
IaIp are endogenous proteins that control excessive inflammatory responses to toxins, infectious organisms, and tissue and organ damage. An inverse correlation between IaIp levels in 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 survival rate.
“We know that IaIp are rapidly consumed and cleared from circulation during severe sepsis cases, as evidenced by the significantly lower plasma levels of IaIp in newborns suffering from NEC. There is also strong scientific evidence indicating that IaIp play a key role in modulating systemic inflammation,” says Dr. John Moran, Prometic’s chief medical officer. “We believe that systemic IaIp administration to replenish these decreased levels could therefore reduce the severity of inflammation and resulting tissue injury experienced by NEC patients.”
Necrotizing enterocolitis is the most common acquired gastrointestinal disease diagnosed in premature neonates and is one of the leading causes of death in neonatal intensive care units. The economic cost of necrotizing enterocolitis is high, accounting for approximately 19 per cent of neonatal expenditures and an estimated $5 billion per year for hospitalizations in the United States alone. If the disease is able to be treated without surgery, the average cost of hospitalization has been estimated at around $73 700, with a length of stay exceeding 22 days more than that for other premature infants. However, if surgical care is required, there is an average additional cost of $186 200, and infants require a length of stay 60 days longer than other premature infants.
“IaIp are just another example of rare proteins that could be made accessible to address unmet medical needs through the use of our proprietary plasma purification platform,” comments Pierre Laurin, Prometic’s president and chief executive officer. “The sequential addition of these rare proteins is key to our strategy of building a unique portfolio of innovative therapeutics targeting unmet medical needs while generating substantial revenue levels with each liter of plasma purified”.
Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.