On May 3rd, 2018 a joint meeting of the Gastrointestinal Drugs Advisory Committee and the Pediatric Advisory Committee was held to discuss the new drug application (NDA) 209904 for InfaCare Pharmaceutical Corporation’s stannsoporfin. Stannsoporfin is administered via intramuscular injection for the treatment of neonates, 35 weeks of gestational age and older, with indicators of hemolysis at risk for developing severe hyperbilirubinemia. Stannsoporfin inhibits the production of bilirubin from the breakdown of red blood cells and therefore reduces total serum bilirubin levels. This can avoid the need to use phototherapy – the current standard of care when the body cannot eliminate bilirubin via the liver.
The sponsor presented data from 15 studies supporting the safety and efficacy of the drug in neonates and determined that a 4.5 mg/kg dose of stannsoporfin, with or without phototherapy, effectively treats neonatal hyperbilirubinemia and maintains a favorable safety profile. The sponsor also discussed the risks that accompanied the use of phototherapy and its potential side effects for neonates.
Members of the advisory committee expressed concerns with the drug’s effect on neurodevelopment and its overall clinical benefit. Some members indicated that incorporation into the FDA’s REMS program would not be enough to safely put the drug on the market. Most of the panel felt the combined safety and efficacy of the drug was not enough to support approval. Other concerns included study parameters, small sample sizes and a lack of ability to make comparisons due to the patient selection. The committee voted 21 to 3 against approving the medication. The agency is not obligated to follow its panel’s recommendation, but normally does so.
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This post was authored by Sami Mourad. Sami is a summer intern at ISS and a 3rd year pharmacy student at the Rutgers University Ernest Mario School of Pharmacy