FDA Clears Sacubitril/Valsartan for Children With Heart Failure

Megan Brooks

October 03, 2019

The US Food and Drug Administration (FDA) has approved sacubitril/valsartan (Entresto, Novartis) for the treatment of symptomatic heart failure (HF) with systemic left ventricular systolic dysfunction in children 1 year and older.

Sacubitril/valsartan was approved in the United States in 2015 to reduce the risk for cardiovascular (CV) death or HF hospitalization in adult patients with chronic heart failure (NYHA class II to IV) and reduced ejection fraction (HFrEF).

Children diagnosed with systolic heart failure face significant morbidity and mortality; about half require a heart transplant before age 5 and almost one-third die or require a transplant within a year of diagnosis, Novartis said in a news release announcing the expanded indication.

The pediatric indication is based on an analysis at 12 weeks from the 52-week PANORAMA-HF trial, which showed that treatment with sacubitril/valsartan led to reductions in the cardiac biomarker N-terminal pro-B-type natriuretic peptide (NT-proBNP) in children 1 to less than 18 years of age with HF due to systemic left ventricular systolic dysfunction.

Because sacubitril/valsartan improved outcomes and reduced NT-proBNP in adults in the PARADIGM-HF trial, this effect on NT-proBNP was considered a reasonable basis to infer improved cardiovascular outcomes in pediatric patients, according to the company. The safety and tolerability of sacubitril/valsartan in children is consistent with that observed in adults.

"PANORAMA-HF is ongoing to secure 52-week follow-up data for the full study population. We remain committed to trial completion to generate further evidence on how Entresto impacts the clinical course of pediatric HF," David Soergel, MD, global head of cardiovascular, renal, and metabolic drug development at Novartis, said in the news release.

"Pediatric heart failure is extremely serious and carries a substantial burden. Given the significant unmet need for heart failure treatments that are proven safe and effective in pediatric patients, this approval and the availability of a new treatment option is great news for these children and their families," Andrea Baer, executive director, Mended Little Hearts, said in the release.

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