FDA approves Perflutren Lipid Microsphere for pediatric patients with suboptimal echocardiograms

FDA approves Perflutren Lipid Microsphere for pediatric patients with suboptimal echocardiograms | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA has approved a supplemental New Drug Application (sNDA) for Perflutren Lipid Microsphere (DEFINITY; Lantheus Holdings) injectable suspension for use in pediatric patients with suboptimal echocardiograms as an ultrasound enhancing agent.

The approval, according to a press release from Lantheus Holdings, will provide health care professionals with a tool to opacify the left ventricular chamber to better identify the left ventricular endocardial border.

"While [Perflutren Lipid Microsphere] 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, chair, Department of Radiology, 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 federal agency approved Perflutren Lipid Microsphere following data from 3 pediatric clinical trials:

• The Golding study (40 patients aged 1 month to 17 years with significant cardiovascular disorders).
• The Fine study (36 patients aged 10 to 21 years with a diagnosis of known congenital or acquired heart disease or suspected cardiac disease).
• The Kutty study (113 patients [retrospective] aged 5 to 21 years with a diagnosis of known congenital or acquired heart disease or suspected cardiac disease).

Data from the Golding study demonstrated that left ventricular opacification with Perflutren Lipid Microsphere was successful and that ultrasound contrast provided information to guide management (cumulative doses ranging from 6 µL/kg to 20 µL/kg).

The ability to detect wall motion abnormalities and the ability to perform ejection fraction determinations were improved in 70% and 80% of patients in the Golding study, respectively.

Left ventricular opacification was observed in all patients in the Fine and Kutty studies. In the Kutty study, Lantheus Holdings stated, "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."

Regional wall motion abnormalities were identified in 3 patients in the Fine study (8.3%, n = 36) and in 32 patients (88.9%), demonstrated successful myocardial perfusion imaging. Imaging in some patients was complicated by movement and/or insufficient heart rate factors, stated Lantheus Holdings.

Across all trials, all reported adverse events were brief, mild, and reversible without intervention. No adverse events were reported among study participants in the Golding study.

Perflutren Lipid Microsphere is contraindicated in patients with known or suspected hypersensitivity to perflutren lipid microsphere or its components, such as polyethylene glycol, the radiopharmaceutical-focused company stated.

Reference:

Lantheus anounces the FDA approval of DEFINITY (Perflutren Lipid Microsphere) for pediatric patients. Lantheus Holdings. Press release. March 4, 2024. Accessed March 4, 2024. https://lantheusholdings.gcs-web.com/news-releases/news-release-details/lantheus-announces-fda-approval-definityr-perflutren-lipid