Valsartan / sacubitril

Extended indication	Extension of indication to include treatment of children and adolescents aged one year or older with chronic heart failure with left ventricular systolic dysfunction.
Therapeutic value	Possible added value
Registration phase	Registered and reimbursed

Product

Active substance	Valsartan / sacubitril
Domain	Cardiovascular diseases
Reason of inclusion	Indication extension
Main indication	Other medication for cardiovascular diseases
Extended indication	Extension of indication to include treatment of children and adolescents aged one year or older with chronic heart failure with left ventricular systolic dysfunction.
Proprietary name	Entresto
Manufacturer	Novartis
Route of administration	Oral
Therapeutical formulation	Granulate
Budgetting framework	Extramural (GVS)
Additional remarks	The CHMP adopted the addition of a new pharmaceutical form associated with two new strengths (Entresto 6mg/6mg and15mg/16mg granules in capsule for opening) and a new indication to include treatment of children and adolescents with heart failure. The CHMP also adopted an extension to the existing indication for Entresto film coated tablets.

Registration

Registration route	Centralised (EMA)
Particularity	New therapeutical formulation
ATMP	No
Submission date	July 2022
Expected Registration	May 2023
Orphan drug	No
Registration phase	Registered and reimbursed
Additional remarks	Positieve CHMP 30 maart .

Therapeutic value

Current treatment options	Enalapril
Therapeutic value	Possible added value
Substantiation	In het document van de EMA is een daling analoog aan de volwassen studie te zien. Secundaire uitkomsten zijn tevens beter in de groep die behandeld is. Verondersteld wordt dat, omdat deze daling vergelijkbaar is met volwassenen de effectiviteit vergelijkbaar is.
Dosage per administration	6 mg/6 mg - 15 mg/16 mg
References	NCT02678312
Additional remarks	Nieuwe toedieningsvorm: 6mg/6mg granules in capsule for opening and15mg/16mg granules in capsule for opening.

Expected patient volume per year

Patient volume	33 Market share is generally not included unless otherwise stated.
References	Hypertrophic Cardiomyopathy With Left Ventricular Systolic Dysfunction Insights From the SHaRe Registry. Peter Marstrand. 2020 (1); Expert opinie (2); CBS (3)
Additional remarks	De prevalentie van een linker ventrikel systolische functiestoornis was 3,7% in de algehele populatie (1). Ongeveer één op de 100.000 kinderen heeft de diagnose (2). Er zijn 3,3 miljoen kinderen tot 18 jaar in Nederland. Dit betekent dat er ongeveer 33 kinderen in aanmerking zullen komen.

Patient volume

33

Market share is generally not included unless otherwise stated.

References

Hypertrophic Cardiomyopathy With Left Ventricular Systolic Dysfunction Insights From the SHaRe Registry. Peter Marstrand. 2020 (1); Expert opinie (2); CBS (3)

Additional remarks

De prevalentie van een linker ventrikel systolische functiestoornis was 3,7% in de algehele populatie (1). Ongeveer één op de 100.000 kinderen heeft de diagnose (2). Er zijn 3,3 miljoen kinderen tot 18 jaar in Nederland. Dit betekent dat er ongeveer 33 kinderen in aanmerking zullen komen.

Expected cost per patient per year

Additional remarks	Fabrikant geeft aan dat de prijs nog niet bekend is.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.