Novartis announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Entresto (sacubitril/valsartan) for paediatric patients aged one year and above with symptomatic chronic heart failure (HF).

Key opinion leaders interviewed by GlobalData have stated that a big issue among HF patients is that they do not receive therapy at optimal doses, leaving them under-dosed. GlobalData believes the CHMP’s positive opinion is likely to shed more light on Entresto’s ability to provide children with HF with an age-appropriate drug formulation, enabling accurate and convenient drug administration.

Entresto is seen as a promising new addition for the paediatric population. If it receives approval, Entresto will be the first therapy to be approved for symptomatic chronic HF for patients aged one year and above in the EU. This is significant since up to 33% of paediatric cardiac hospital admissions are associated with HF.

The positive CHMP recommendation is based on data from Phase III programmes. Entresto was seen as a revolutionary advancement in the HF field, due to its demonstrated efficacy in the Phase III PARADIGM-HF trial in reducing the number of deaths due to cardiovascular causes, HF hospitalisations and all-cause mortality compared to angiotensin-converting enzyme inhibitors.

The Phase III PANORAMA-HF trial was the largest trial for paediatric HF. The trial showed reduced N-terminal pro-brain natriuretic peptide; elevated levels of this peptide cause reduced pumping capacity in the heart and increased severity of HF.

Entresto was launched in the US in July 2015, and subsequently across the five major European markets (5EU: France, Germany, Italy, Spain and the UK) in 2016. Valsartan blocks the vasoconstrictor and aldosterone-secreting effects of angiotensin II by selectively blocking the binding of angiotensin II to the AT1 receptor in many tissues. Sacubitril is an inhibitor of neutral endopeptidase inhibitor.

The goals of treatment for chronic HF are to relieve symptoms, prevent HF-associated hospitalisations, slow disease progression and improve survival. Current therapies on the market are well-established. Angiotensin-converting enzyme inhibitors, angiotensin receptor blockers and beta blockers are all used within the HF landscape regardless of the type of HF. All these drugs have clinical limitations. There is a strong opportunity in this disease space for new agents that could further reduce the incidence of HF-associated hospitalisations and mortality.