Entresto (sacubitril/valsartan) is intended for treating symptomatic chronic heart failure with left ventricular systolic dysfunction in patients who are aged from 1 to <18 years.
Novartis global head for cardiovascular, renal and metabolism development unit David Soergel said: “Many children diagnosed with heart failure will need a heart transplant before they turn five or in the worst cases almost a third will die within a year. However, the disease is largely managed with treatments approved for adults, based on data from adult studies.
“This positive opinion paves the way towards Novartis being able to provide children diagnosed with heart failure due to left ventricular systolic dysfunction in the EU and their families with an effective therapy in an age-appropriate formulation, helping to address what is a major unmet need.”
This positive opinion has been given based on final data from the 52-week Phase III PANORAMA-HF trial, claimed to be the largest pediatric heart failure study ever carried out, and extrapolation of adult heart failure data from the Phase III PARADIGM-HF trial to paediatric patients.
Pediatric heart failure is an important reason for morbidity and mortality in childhood.
Up to 33% of all paediatric cardiac admissions are associated with heart failure.
Novartis stated that children whose hospitalisation is due to heart failure can have over a 20-fold increase in the risk of death.
Entresto has been given approval in the EU since 2015 in adult patients to treat symptomatic chronic heart failure with reduced ejection fraction (HFrEF).
In the US, Entresto is indicated to cut down the risk of cardiovascular death and hospitalisation for heart failure in adult patients with chronic heart failure.
It has given been approval since October 2019 to treat childhood heart failure because of left ventricular systolic dysfunction.