The company gains a therapy for treating jaundice in infants with the acquisition.
Jaundice, also known as infantile hyperbilirubinemia, affects as many as 750,000 infants born in the US each year. While it is a common condition in newborns, if traditional treatments are ineffective, severe jaundice can lead to permanent neurological damage (brain damage) and death in rare cases. Jaundice occurs when there are higher than normal levels of bilirubin in the blood. In newborns is occurs due to accelerated breakdown of red blood cells and can lead to severe bilirubin levels. At these high levels, there is a risk that the bilirubin can enter the brain, a condition referred to as acute bilirubin encephalopathy. Immediate treatment is required to prevent progression to kernicterus, a rare condition associated with severe and permanent brain damage.
A new drug developed by InfaCare, a privately held specialty pharmaceutical company focused on development and commercialization of proprietary pharmaceuticals for neonatal and pediatric patient populations, will now be pursued by specialty pharma company Mallinckrodt following its acquisition of InfaCare. The deal involves an upfront payment to InfaCare of $80 million and up to $345 million in regulatory and sales milestones payments.
InfaCare’s New Drug Application (NDA) for stannsoporfin was accepted by FDA in July 2016 based on the results of tow Phase II(b) trials. FDA also granted stannsoporfin its Fast Track designation. Approval of the drug is expected in the first half of 2018.
When phototherapy, the common standard treatment for jaundice, fails, invasive treatment options must be used—typically blood exchange transfusion but in some cases intravenous immunoglobulin infusions (IVIG), which are complex and lengthy procedures. All of these methods involve removal of excess bilirubin after it is produced. Often severe jaundice requires multiple treatments.
Stannsoporfin operates by a novel mechanism of action that involves the inhibition of bilirubin production. According to Mallinckrodt, stannsoporfin has been shown to have a good safety profile when compared to a placebo and is easy to administer via intramuscular injection. If it receives approval, stannsoporfin will be the “first and only pharmacologic treatment indicated for treatment of newborns at risk for developing severe infantile jaundice in the US,” according to the company.
"We look forward to bringing this much-needed treatment option to babies at greatest risk for the consequences of this condition,” said Steven Romano, MD, Chief Scientific Officer and Executive Vice President of Mallinckrodt. Added Mark Trudeau, Chief Executive Officer and President of Mallinckrodt: "We believe stannsoporfin has the potential to significantly alter the treatment paradigm for infants with this condition which, if unchecked, can have devastating impact to the patient.” Dan Burns, President and Chief Executive Officer, InfaCare further commented that “We believe stannsoporfin has the potential to help thousands of infants whose severe jaundice is unresolved by current treatments. We're also excited by the additional development capability and commercial reach that can be gained by becoming part of Mallinckrodt. Together I'm confident we can successfully bring this important treatment to market."
If approved, Mallinckrodt expects stannsoporfin to “be a highly effective therapy used for near- and full-term infants at risk of developing complications associated with severe jaundice,” potentially “reduce the number of newborns advancing to bilirubin levels requiring more intrusive, less specific therapies” and possibly “decrease the risks associated with other treatments (e.g., bilirubin rebound) and the risk of prolonged and/or severe bilirubin elevation, which can impact central nervous system development."
The acquisition of InfaCare and stannsoporfin expands Mallinckrodt's pediatric offerings, diversifying its hospital portfolio pipeline with this differentiated, highly durable product, according to the company, which estimates there are 150,000 to 275,000 annually babies worldwide that could benefit from the drug. “The addition of this highly durable, unique developmental asset to our growing hospital business is an excellent example of Mallinckrodt's investment strategy," said Trudeau.