If FDA approved, it would be the first-in-class pharmacologic treatment for hyperbilirubinemia in newborns that contains a novel mechanism of action inhibiting bilirubin production at the source.
The US Food and Drug Administration’s (FDA) Gastrointestinal Drugs Advisory Committee and Pediatric Advisory Committee recommended in a 21 to 3 vote that the risk-benefit profile of stannsoporfin does not support the approval for treatment of newborns ≥35 weeks of gestational age with indicators of hemolysis at risk of developing hyperbilirubinemia, announced Mallinckrodt.
The committee voted that there was not sufficient evidence to support the effectiveness of stannsoporfin for the proposed indication.
The company will work closely with the FDA as the review process continues to evaluate alternatives for the development program.
Stannsoporfin is a heme oxygenase inhibitor currently under investigation for treatment of newborns ≥35 weeks of gestational age at risk of developing severe jaundice. If approved, stannsoporfin would be the first-in-class pharmacologic treatment for hyperbilirubinemia in newborns that contains a novel mechanism of action inhibiting bilirubin production at the source.
The safety and effectiveness have not yet been established by the FDA.
Neonatal jaundice occurs in about 60% of babies and most often goes away without treatment, however, in some cases, the newborn isn’t able to clear excessive levels of bilirubin that results in an imbalance, which leads to hyperbilirubinemia.
If hyperbilirubinemia is left untreated, the condition may lead to neurologic complications and potentially irreversible brain damage.
Current standard of care treatment includes phototherapy, which targets excess bilirubin that has accumulated in the newborn’s bloodstream.
In December 2016, the FDA also granted stannsoporfin fast track designation. Post-approval commitments required would include conducting trials in pre-term infants less than 35 weeks gestational age as part of the pediatric requirements. If approved, the drug will have substantial durability both as a new chemical entity and through its intellectual property which is valid until 2032.