Valsartan / sacubitril Medicine 07 December 2022 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 Total cost Registration phase Registered and reimbursed Product Active substance Valsartan / sacubitril Domain Cardiovascular diseases Reason of inclusion Indication extension IND 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. Current proprietary name Already available biosimilars / generics Proprietary name Entresto Manufacturer Novartis Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise 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) Type of trajectory Unknown Particularity New therapeutical formulation ATMP No Submission date July 2022 Expected Registration May 2023 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP 30 maart . Therapeutic value Current treatment options Enalapril Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. 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. Duration of treatment Frequency of administration 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. Maximum patient volume for sluice 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 Cost References Additional remarks Fabrikant geeft aan dat de prijs nog niet bekend is. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks