INOmax has been on the market in the
The primary outcome measure of the registry was the number of PT neonates and TNT neonates with a significant response to INOmax, which was defined as at least a 25 percent improvement (decrease) from baseline in oxygenation index or surrogate oxygenation index (OI/SOI) during INOmax treatment. A total of 50 (90.9 percent) PT and 75 (88.2 percent) TNT neonates achieved a ≥25 percent decrease in OI/SOI during treatment with INOmax. Efficacy in the PT group demonstrated non-inferiority (95 percent confidence interval: 0.0267 [-0.0333, 0.0868], with a pre-defined margin of -0.1452). In addition, the proportion of neonates with ≥25 percent decrease in OI/SOI was similar across severity groups with no significant difference in time to improvement between groups.1
"These registry findings help expand our understanding of a potential role of inhaled nitric oxide therapy in preterm infants with hypoxic respiratory failure with pulmonary hypertension," said study author
Persistent pulmonary hypertension of the newborn (PPHN) is a serious and sometimes fatal cardiorespiratory complication of the transition to extra-uterine life.2,3 The registry trial was conducted to examine the utility of INOmax in pre-term neonates. Due to the seriousness of the condition, a randomized controlled trial cannot be conducted in the pre-term neonate population.
Overall, 21 adverse events of special interest were reported in 17 patients, all of which were classified as serious events, and no serious adverse events were attributed to the study drug.
"After ending this registry much earlier than anticipated last year based on positive findings, Mallinckrodt is extremely pleased to be able to share these important data with the healthcare community and add to the body of research and real-world data for this vulnerable patient population," said
About the Observational Registry
More information about the trial can be found here.
The study was a prospective observational registry that collected real world data that described INOmax nitric oxide gas use in clinical practice. As an observational study it did not utilize placebo. Hence, some of the improvement observed in the study could be due to factors other than treatment with INOmax. Similarly, without placebo control it is not possible to understand the magnitude to which patients who experienced limited response to inhaled nitric oxide would have otherwise decompensated without this treatment.
About Persistent Pulmonary Hypertension of the Newborn (PPHN)
PPHN is a serious and sometimes fatal cardiorespiratory complication of the transition to extra-uterine life.2,3 PPHN is a clinical syndrome associated with various neonatal cardiorespiratory diseases, including meconium aspiration, respiratory distress syndrome (hyaline membrane disease), congenital heart disease and congenital hernia.2 Despite the diversity of causes, marked pulmonary vasoconstriction is the central pathophysiologic feature of PPHN.4 The most significant hemodynamic feature in neonates with severe hypoxia is a pulmonary-to-systemic pressure imbalance.5 Treatment in these neonates is directed toward lowering the pulmonary vascular pressure and supporting the systemic circulation.2,4
IMPORTANT SAFETY INFORMATION
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Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; analgesics and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit www.mallinckrodt.com.
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CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS
This release includes forward-looking statements concerning inhaled nitric oxide ("iNO") and the Company's iNO product, including statements with regard to the clinical data generated in the study described above. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues; and other risks identified and described in more detail in the "Risk Factors" section of Mallinckrodt's most recent Annual Report on Form 10-K and other filings with the
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1 Nelin L, Kinsella J, Courtney S, Pallotto E, Tarau E, Potenziano J. Use of Inhaled Nitric Oxide in Preterm vs Term/Near-Term Neonates With Pulmonary Hypertension: Results of the PaTTerN Registry Study. Presented at: Pediatric Academic Societies Annual Meeting;
2 Nair J, Lakshminrusimha S. Update on PPHM: Mechanisms and treatment. Semin Perinatol. 2014;38(2):78-91.
3 Clark RH. The epidemiology of respiratory failure in neonates born at an estimated gestational age of 34 weeks or more. J Perinatol. 2005;25(4):251-257.
4 Wedgwood S, Steinhorn RH, Lakshminrushimha S. Optimal oxygenation and role of free radicals in PPHN. Free Radical Biology and Medicine. 2019;142:97-106.
5 Lakshminrusimha S. The pulmonary circulation in neonatal respiratory failure. Clin Perinatol. 2012;39(3):655-683.
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