Extended indication Adults and Children Suffering from distal Renal Tubular Acidosis (dRTA)
Therapeutic value No judgement
Registration phase Registration application pending

Product

Active substance Kaliumcitraat / kaliumwaterstofcarbonaat
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Adults and Children Suffering from distal Renal Tubular Acidosis (dRTA)
Manufacturer Advicenne
Mechanism of action Other
Route of administration Oral
Therapeutical formulation Granulate
Budgetting framework Extramural (GVS)
Additional remarks Kaliumcitraat / kaliumwaterstofcarbonaat heeft een alkaliserende werking zodat de zuurtegraad in de patiënt wordt teruggebracht.

Registration

Registration route Centralised (EMA)
Particularity New therapeutical formulation
Submission date November 2019
Expected Registration December 2020
Orphan drug Yes
Registration phase Registration application pending

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 judgement
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 continuous
Frequency of administration 2 times a day
References Scrip

Expected patient volume per year

Patient volume

< 600

Market share is generally not included unless otherwise stated.

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 (EU)." 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

There is currently nothing known about the expected cost.

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.