Lantheus Announces the FDA Approval of DEFINITY® (Perflutren Lipid Microsphere) for Pediatric Patients

Rhea-AI Summary

Lantheus Holdings, Inc. announces FDA approval for DEFINITY as an ultrasound enhancing agent for pediatric patients with suboptimal echocardiograms. The approval marks a significant advancement in pediatric medicine, providing healthcare professionals with a valuable diagnostic tool. Clinical studies have confirmed the efficacy and safety of DEFINITY in pediatric patients, offering improved outcomes and important diagnostic information.

Insights

Medical Research Analyst

The FDA approval of DEFINITY for pediatric use marks a significant development in the field of diagnostic imaging. The clinical studies cited demonstrate a clear benefit in the visualization of the left ventricular chamber, which is crucial for accurate diagnosis and treatment planning in pediatric cardiology. The ability to better delineate the left ventricular endocardial border can lead to more precise assessments of conditions like congenital heart defects, which are among the most common birth defects.

From a research perspective, the safety profile of DEFINITY is noteworthy. The absence of serious adverse events in clinical trials is reassuring for clinicians and parents alike. This could potentially lead to increased adoption rates in pediatric echocardiography. Furthermore, the efficacy in improving the detection of wall motion abnormalities and ejection fraction determinations aligns with the goals of pediatric cardiologists to provide comprehensive care.

The data from the Golding, Fine and Kutty studies indicate that DEFINITY can be a game-changer for pediatric heart patients. It is important to consider, however, that while the studies showed positive results, the long-term impact on patient outcomes and the healthcare system remains to be seen. Continuous post-marketing surveillance will be crucial to monitor the real-world safety and effectiveness of DEFINITY in this new patient population.

Pediatric Cardiologist

The expanded indication of DEFINITY for pediatric patients addresses a significant unmet need in pediatric echocardiography. Pediatric cardiologists often face challenges in obtaining clear images due to the small size of patients and rapid heart rates. DEFINITY's ability to opacify the left ventricular chamber enhances the quality of echocardiographic images, which is paramount in diagnosing and managing pediatric cardiac conditions.

The clinical impact of this FDA approval is substantial. For pediatric patients with suboptimal echocardiograms, DEFINITY offers a non-invasive method to improve diagnostic accuracy. This can lead to better-informed clinical decisions and potentially reduce the need for more invasive procedures such as cardiac catheterization, which carry higher risks and costs.

While the clinical trials referenced provide strong support for DEFINITY's use in this population, it is essential for pediatric cardiologists to weigh the benefits against any potential risks, even if they are minimal. The improvement in diagnostic capabilities must be balanced with careful patient monitoring, especially in a vulnerable pediatric population.

Healthcare Economist

The FDA approval of DEFINITY for pediatric use has implications for the healthcare economy. Diagnostic enhancements that improve accuracy and reduce the need for additional testing can lead to cost savings for healthcare systems. By potentially eliminating the need for further diagnostic procedures, which can be invasive and expensive, DEFINITY could contribute to more efficient use of healthcare resources.

In terms of market impact, Lantheus Holdings, Inc. could see an increase in demand for DEFINITY, given its new pediatric application. This expansion into the pediatric market could lead to revenue growth for the company, as well as increased market share in the ultrasound contrast agent segment. It's important to analyze how this approval may affect the company's financial performance and its position within the competitive landscape of radiopharmaceuticals and ultrasound enhancing agents.

However, broader economic considerations include the accessibility and affordability of DEFINITY for pediatric patients across different healthcare systems. The value proposition of DEFINITY must be analyzed in the context of healthcare reimbursement policies and potential barriers to access, which can vary significantly by region.

DEFINITY is the #1 utilized ultrasound enhancing agent in the U.S. for patients with suboptimal echocardiograms

BEDFORD, Mass., March 04, 2024 (GLOBE NEWSWIRE) -- Lantheus Holdings, Inc. (“Lantheus”) (NASDAQ: LNTH), the leading radiopharmaceutical-focused company committed to enabling clinicians to Find, Fight and Follow^® disease to deliver better patient outcomes, today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application (sNDA) for DEFINITY^® (Perflutren Lipid Microsphere) as an ultrasound enhancing agent for use in pediatric patients with suboptimal echocardiograms. This approval represents a significant step forward in pediatric medicine, providing healthcare professionals with a valuable tool to opacify the left ventricular chamber and better identify the left ventricular endocardial border. Currently, DEFINITY is the most utilized, extensively studied, and a trusted diagnostic ultrasound enhancing agent in the U.S.^1-3

“The expanded indication for DEFINITY is a testament to the rigorous research and development efforts undertaken by Lantheus to address unmet medical needs of pediatric patients,” said Jean-Claude Provost, MD, Chief Medical Officer. “We are proud that this approval will extend the benefits of DEFINITY to healthcare professionals who work with these young individuals.”

Clinical studies have substantiated the efficacy and safety of DEFINITY in pediatric patients.

"While DEFINITY has long demonstrated its effectiveness in providing better outcomes in adults over the past two decades, this new FDA decision offers a critical diagnostic tool for pediatric heart patients and their families,” said Kassa Darge, MD, PhD, Radiologist-in-Chief and Chair of the Department of Radiology at Children’s Hospital of Philadelphia. “This approval will offer a valuable alternative to pediatric cardiologists trying to do imaging work up in challenging pediatric cardiac cases.”

The FDA decision was based on usage data from three pediatric clinical trials conducted with DEFINITY: the Golding study, which evaluated 40 patients, ranging from 1 month to 17 years of age, with significant cardiovascular disorders, including heart transplant, Kawasaki disease or congenital cardiovascular anomaly; the Fine study, which evaluated 36 patients, ranging from 10 to 21 years of age, who had previously undergone a heart transplant; and the Kutty study, a retrospective medical record review of 113 pediatric patients, ranging from 5 to 21 years, who had a diagnosis of known congenital or acquired heart disease or suspected cardiac disease. These studies evaluated the use of DEFINITY in a total of 189 patients (107 (56.5%) were male and 82 (43.5%) were female).^4-6  

The Golding study, together with supportive data from the Fine and Kutty studies, demonstrate that left ventricular opacification with DEFINITY (cumulative doses ranging from 6 µL/kg to 20 µL/kg) is successful in pediatric patients and that ultrasound contrast provides important information to guide management in such patients. In the Golding study, both the ability to detect wall motion abnormalities and the ability to perform ejection fraction determinations were improved in the majority (70% and 80%, respectively) of subjects. The Kutty and Fine studies each reported successful left ventricular opacification in all participants. In the Kutty study, wall motion and/or myocardial perfusion wall motion abnormalities were identified in 13 (11.5%) of the patients (all identified during stress testing), and the number of left ventricular segments visualized improved from 13 ± 1 per patient without contrast to 16 ± 1 segments per patient with contrast across all patients. The Fine study identified regional wall motion abnormalities detected in three patients (8.3%, n=36), and demonstrated successful myocardial perfusion imaging in 32 patients (88.9%), with imaging in some patients complicated by movement and/or insufficient heart rate factors.^4-6     

All reported adverse events across all trials were mild, brief and reversible without intervention. Specifically, the Golding study reported no AEs among study participants; the Fine study reported that 33% of patients experienced mild AEs (palpitations, headache, nausea and shortness of breath) attributed to stress test procedures and not as a result of DEFINITY administration; and the Kutty study reported 11% of patients experienced AEs with no differences between DEFINITY and those patients who did not receive DEFINITY in frequencies of adverse events (chest/back/neck pain, headache, fatigue, dizziness, shortness of breath).^4-6  

About DEFINITY^® 
DEFINITY Vial for (Perflutren Lipid Microsphere) Injectable Suspension is an ultrasound enhancing agent approved for use in patients with suboptimal echocardiograms (see Indications and Important Safety Information below and find full Prescribing Information at www.definityimaging.com).⁶ DEFINITY is the most utilized, extensively studied and a trusted ultrasound enhancing agent with more than 20 years in the market.^1-3

DEFINITY^® Vial for (Perflutren Lipid Microsphere) Injectable Suspension

INDICATIONS
DEFINITY^® is indicated, after activation, for use in adult and pediatric patients with suboptimal echocardiograms to opacify the left ventricular chamber and to improve the delineation of the left ventricular endocardial border.

CONTRAINDICATIONS
DEFINITY^® is contraindicated in patients with known or suspected hypersensitivity to perflutren lipid microsphere or its components, such as polyethylene glycol (PEG) [see Warnings and Precautions (5.2) and Description (11)].

IMPORTANT SAFETY INFORMATION

WARNING: Serious Cardiopulmonary Reactions Serious cardiopulmonary reactions, including fatalities, have occurred uncommonly during or following perflutren-containing microsphere administration. Most serious reactions occur within 30 minutes of administration. Assess all patients for the presence of any condition that precludes DEFINITY® administration [see Contraindications (4)]. Always have resuscitation equipment and trained personnel readily available [see Warnings and Precautions (5.1)].

In post-marketing use, rare but serious cardiopulmonary or hypersensitivity reactions have been reported during or shortly following perflutren and PEG-containing microsphere administration [see Adverse Reactions (6)]. The risk for these reactions may be increased among patients with unstable cardiopulmonary conditions and/or with pre-existing PEG hypersensitivity [see Adverse Reactions (6.2)]. It is not always possible to reliably establish a causal relationship to drug exposure due to the presence of underlying conditions.

Patients with sickle cell disease may be at higher risk of painful crisis and DEFINITY^® should be administered with caution.

Healthcare providers and pediatric specialists are encouraged to review the updated prescribing information for DEFINITY, when available, to incorporate the expanded indication into their clinical practice.

Please see accompanying full Prescribing Information for DEFINITY^®, including boxed WARNING regarding serious cardiopulmonary reactions, on www.definityimaging.com.

About Lantheus
Lantheus is the leading radiopharmaceutical-focused company, delivering life-changing science to enable clinicians to Find, Fight and Follow disease to deliver better patient outcomes. Headquartered in Massachusetts with offices in New Jersey, Canada and Sweden, Lantheus has been providing radiopharmaceutical solutions for more than 65 years. For more information, visit www.lantheus.com.

Safe Harbor for Forward-Looking and Cautionary Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, that are subject to risks and uncertainties and are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by their use of terms such as “aim,” “will” and other similar terms. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to materially differ from those described in the forward-looking statements. Readers are cautioned not to place undue reliance on the forward-looking statements contained herein, which speak only as of the date hereof. Lantheus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. Risks and uncertainties that could cause Lantheus’ actual results to materially differ from those described in the forward-looking statements are discussed in Lantheus’ filings with the Securities and Exchange Commission (including those described in the Risk Factors section in Lantheus’ Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q).

¹Data on file, Lantheus.
²Embase and Medline Search, May 2018; Data on file, Lantheus.
³©2021 Millennium Research Group, Inc. All rights reserved. Reproduction, distribution, transmission or publication is prohibited. Reprinted with permission.
⁴Fine, N. M., et. al (2021). Myocardial Contrast Echocardiography for Cardiac Allograft Vasculopathy: Feasibility of Real-Time Myocardial Contrast Echocardiography to Detect Cardiac Allograft Vasculopathy in Pediatric Heart Transplant Recipients. Journal of the American Society of Echocardiography.
⁵Kutty, S., et al (2016). Safety and Efficacy of Cardiac Ultrasound Contrast in Children and Adolescents for Resting and Stress Echocardiography. Journal of the American Society of Echocardiography.
⁶ DEFINITY^® (Package Insert), North Billerica, MA, Lantheus Medical Imaging, Inc.

Contacts:

Lantheus
Mark Kinarney
Vice President, Investor Relations
978-671-8842
ir@lantheus.com

Melissa Downs
Senior Director, External Communications
646-975-2533
media@lantheus.com

FAQ

What is the FDA approval announced by Lantheus Holdings, Inc. related to the ultrasound enhancing agent for pediatric patients?

The FDA approved the supplemental new drug application (sNDA) for DEFINITY (Perflutren Lipid Microsphere) as an ultrasound enhancing agent for use in pediatric patients with suboptimal echocardiograms.

Who is the Chief Medical Officer mentioned in the press release?

Jean-Claude Provost, MD is the Chief Medical Officer mentioned in the press release.

What were the key findings of the Golding, Fine, and Kutty studies related to the usage of DEFINITY in pediatric patients?

The studies showed successful left ventricular opacification with DEFINITY, improvement in wall motion abnormalities detection, and successful myocardial perfusion imaging in pediatric patients.

What were the reported adverse events in the pediatric clinical trials conducted with DEFINITY?

Reported adverse events were mild, brief, and reversible without intervention, with symptoms like palpitations, headache, nausea, and shortness of breath.