Kaliumcitraat / kaliumwaterstofcarbonaat Medicine 12 June 2018 Extended indication Sibnayal is indicated for the treatment of distal renal tubular acidosis (dRTA) in adults, adolescents and children aged one year and older. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Kaliumcitraat / kaliumwaterstofcarbonaat Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Sibnayal is indicated for the treatment of distal renal tubular acidosis (dRTA) in adults, adolescents and children aged one year and older. Current proprietary name Already available biosimilars / generics Proprietary name Sibnayal Manufacturer Advicenne Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Kaliumcitraat/kaliumwaterstofcarbonaat heeft een alkaliserende werking zodat de zuurtegraad in de patiënt wordt teruggebracht. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New therapeutical formulation ATMP Unknown Submission date November 2019 Expected Registration April 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Alkaliserende middelen om de zuurtegraad van het lichaam in stand te houden zoals natriumbicarbonaat, natriumcitraat en kaliumcitraat. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Huidige behandeling moet iedere 4-6 uur worden ingenomen. Deze formulering werkt 12 uur en hoeft dus maar 2 maal per dag te worden ingenomen en is smaakloos. Met name gericht op kindvriendelijke toediening. Duration of treatment Frequency of administration 2 times a day Dosage per administration References Scrip Additional remarks Expected patient volume per year Patient volume < 600 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References EMA, expert opinie Additional remarks EMA: "At the time of designation, distal renal tubular acidosis affected approximately 2.1 in 10,000 people in the European Union." Dit zou neerkomen op 3.570 patiënten in Nederland. Volgens experts is de verwachting dat er mogelijk zo'n 50-100 kinderen in aanmerking komen voor deze behandeling en 250-500 volwassen, rekening houdend met het feit dat het in de urologie therapie is om steenvorming tegen te gaan. Schatting maximaal 600 patiënten. Expected cost per patient per year Cost References Additional remarks De prijs is op dit moment niet bekend maar is mogelijk hoger dan de losse componenten die beschikbaar zijn. 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