The U.S. Food and Drug Administration (FDA) recently granted Pittsburgh-based Noveome Biotherapeutics the Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for its ST266 drug.
The designations are for the treatment of Necrotizing Enterocolitis (NEC).
The RPDD is for serious and life-threatening diseases affecting children 18 years old or younger, and ODD is for diseases that impact fewer than 200,000 people in the United States.
ST266 is a cell-free sterile biologic solution containing proteins. In preclinical studies, the drug resulted in various anti-inflammatory and neuroprotective responses.
NEC is caused by inflammation of the intestines and can result in an infection that often requires emergency surgery. The disease is seen primarily in premature and very low birth weight babies and has a 30 percent mortality rate. Surviving babies often have life-long intestinal complications and are at increased risk for neurodevelopmental delays.
Annually, there are between 4,000 and 6,000 cases and more than $5 billion in neonatal intensive care unit expenditures. There is no FDA-approved treatment.
Noveome Biotherapeutics is a clinical-stage biopharmaceutical company focused on developing next-generation biologic therapeutics for inflamed or damaged tissues.
The company recently completed a Phase 1 study evaluating the safety of intravenously administered ST266 in treating systemic inflammation associated with COVID-19.