Kernicterus

The test is designed to work on the medical device company’s FINDER platform, which leverages the company’s digital microfluidics (DMF) technology, to deliver the G6PD enzyme activity results within 15 minutes The G6PD test is powered by DMF technology. (Credit: PRNewswire/BAEBIES, INC.) Baebies has obtained the US Food and Drug Administration (FDA) 510(k) approval for its rapid, point-of-care test to detect glucose-6-phosphate dehydrogenase (G6PD) deficiency. The test is designed to work on the medical device company’s FINDER platform and comes with a compact instrument and a disposable cartridge. It uses only one drop (50µL) of blood sample and delivers results within 15 minutes. The FINDER platform test displays the G6PD enzyme activity results in units per gram of haemoglobin and adjusted male median values. In December 2019, Baebies secured the CE mark approval for the FINDER platform, which leverages the company’s digital microfluidics (DMF) technology. DMF technology involves a method of manipulating small droplets of liquid by electrical control of surface tension on a disposable cartridge. It eliminates the need for mechanical pumps or valves for liquid handling, reduces the sample volume required, and provides rapid diagnostic results, said the company. Baebies co-founder and CEO Richard West said: “This is a significant milestone not only for our company but for patients whose lives can be improved by detection of G6PD deficiency. “FINDER performs rapid testing from just one drop of blood, making testing easier and more accessible. With G6PD as the first FDA-cleared test on our FINDER platform, we look forward to adding many additional types of tests to the versatile and multifunctional platform.” According to the firm, deficiency in the G6PD enzyme is the world’s most common enzyme deficiency and causes premature destruction of red blood cells. The most common clinical symptom related to G6PD deficiency is haemolytic anaemia, which occurs when red blood cells are destroyed faster than the body can replace them. In addition to haemolytic anaemia, G6PD deficiency also causes mild to severe jaundice in newborns and can lead to kernicterus in some cases. Baebies said that the early diagnosis of G6PD deficiency plays important role in enabling paediatric and adult patients to receive clinical care to manage symptoms. Its G6PD deficiency test is the first among several other assays that are currently under development, for use on the FINDER platform. The other assays are being developed to address unmet needs in haematology and infectious disease through multifunctional syndromic testing. A team of researchers at Stanford University School of Medicine, led by professor Vinod K Bhutani, first evaluated the FINDER G6PD assay compared to known reference methods. Bhutani said: “Recent updates to the AAP clinical practice guidelines on hyperbilirubinemia delineate an urgent need to identify G6PD deficient newborns so as to institute timely interventions such that educational, preventive and treatment options are offered soon after birth. “Implementation of this mode of assay in nurseries will allow clinicians to know G6PD enzyme activity before a newborn is discharged from the hospital. In fact, FDA clearance of Baebies’ digital microfluidics assay introduces a new and important diagnostic solution to identify infants with G6PD deficiency.”

August 4, 2017 By Alex Keown , BioSpace.com Breaking News Staff STAINES-UPON-THAMES, United Kingdom – Specialty pharmaceutical company Mallinckrodt plc struck a deal to acquire privately-held InfaCare , a Pennsylvania-based company focused on therapies for neonatal and pediatric patient. Mallinckrodt will fork over up to $425 million for InfaCare’s stannsoporfin, a heme oxygenase inhibitor, used to treat elevated bilirubin levels that can cause severe jaundice in infants. According to terms of the deal, Mallinckrodt will pay $80 million in upfront monies, and up to $345 million in milestone payments. In late July 2016, the U.S. Food and Drug Administration accepted InfaCare’s New Drug Application for stannsoporfin. In December 2016, the FDA also granted stannsoporfin its Fast Track designation, which is expected to accelerate review of the therapy. The FDA is expected to review the drug in the first half of 2018. Jaundice is fairly common in newborns, but severe jaundice, the highly elevated bilirubin levels, can be threatening. If a baby develops severe jaundice, there is a risk of bilirubin passing into the brain, a syndrome called acute bilirubin encephalopathy. Prompt treatment is needed. Persistent, high levels of bilirubin in the brain can progress to kernicterus, a rare condition associated with severe and permanent brain damage. Symptoms include poor feeding, shrill cry, muscle rigidity, markedly arched back with a backwards hyperextension of the neck, seizures, and stupor or coma. Complications may also include hearing loss and death. There are approximately 750,000 cases of jaundice each year in term babies in the United States. Of those, Mallinckrodt estimates there are 70,000 to 125,000 cases of severe jaundice. Severe jaundice requires extended or recurrent treatment, with costs estimated to be approximately $5,000 per patient, according to Mallinckrodt. Trial data shows that stannsoporfin reduced the potential for developing severe jaundice. Data showed stannsoporfin inhibited bilirubin production. The drug also had a strong safety pro to placebo. If approved, this proprietary therapy is expected to be the first and only pharmacologic treatment indicated for treatment of newborns at risk for developing severe infantile jaundice in the U.S., Mallinckrodt said in its announcement. Dan Burns , president and chief executive officer of InfaCare, said stannsoporfin has the potential to help thousands of infants whose severe jaundice is unresolved by current treatments. “Severe hyperbilirubinemia can result in serious complications in infants, including brain damage and, rarely, death,” Steven Romano , Mallinckrodt’s chief scientific officer, said in a statement. “We look forward to bringing this much-needed treatment option to babies at greatest risk for the consequences of this condition.” Being absorbed by Mallinckrodt will provide InfaCare with a greater reach for its products, Burns said in a statement. Mallinckrodt estimates that the number of severe jaundice cases in term babies globally is between 150,000 and 275,000 per year. In addition to seeking regulatory approval in the United States, Mallinckrodt intends to seek approval in other global markets. Shares of Mallinckrodt are up this morning, trading at $42.15 as of 11:32 a.m.