Rare Daily Staff

The U.S Food and Drug Administration has granted a Rare Pediatric Disease Designation to Prometic Life Sciences for its experimental therapeutic for the treatment of necrotizing enterocolitis, an inflammatory bowel condition that affects predominantly premature infants and can result in an overwhelming infection and death.

NEC accounts for approximately 19 percent of the United States’s annual neonatal medical expenditures, as well as an estimated $5 Billion in annual hospitalization costs in the United States.

Prometic is developing an Inter-Alpha-Inhibitor-Protein, or IaIp, therapy for NEC. IaIp are endogenous proteins that control excessive inflammatory responses to toxins, infectious organisms, tissue and organ damage.  Prometic derives IaIp from purified blood plasma.

An inverse correlation between IaIp levels in blood plasma and disease severity / mortality has been demonstrated in humans with sepsis. The company said in animal models it has shown that the supplementation of IaIp significantly increased the study subjects’ survival rates. The FDA previously granted Prometic Orphan Drug Designation for the therapy.

“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 Pierre Laurin, president and CEO of Prometic. “The combination of pediatric and orphan drug designations provides us with valuable commercial incentives to continue expanding our pipeline of orphan drugs.”

Rare Pediatric Disease Designation makes a company eligible for a Rare Pediatric Disease Priority Review Voucher if the therapy granted the designation is approved. The designation was created to encourage the development of treatments for rare pediatric diseases. The vouchers are potentially lucrative because they are transferable and can be used to accelerate the review of large market therapeutics. The priority review voucher entitles the holder to priority review of a single New Drug Application or Biologics License Application.  Most recently Ultragenyx sold a rare pediatric priority review voucher it received for its MPSVII enzyme replacement therapy for $130 million.

March 5, 2018
Photo: Pierre Laurin, president and CEO of Prometic