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Encaleret

Extended indication Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Encaleret
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Manufacturer BridgeBio Pharma
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional remarks Calcium-sensing receptor antagonist; Osteogenesis stimulant; Parathyroid hormone receptor agonist.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2024
Expected Registration 2025
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Primary completion datum december 2023.

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation Op dit moment loopt de fase 3 studie. Voor nu is enkel te zeggen dat het mechanistisch aangrijpt op de juiste plek wat betreft pathogenese. Nieuwe resultaten worden verwacht in 2024.
References NCT05680818

Expected patient volume per year

Patient volume

243 - 663

Market share is generally not included unless otherwise stated.
References Gunn IR et al. Clinical and laboratory features of calcium‐sensing receptor disorders: a systematic review. 2004 (1); Dershem R et al. Familial hypocalciuric hypercalcemia type 1 and autosomal‐dominant hypocalcemia type 1: prevalence in a large healthcare population. Am J Hum Genet. 2020 (2).
Additional remarks ADH1 heeft een geschatte prevalentie van 1 per 70.000 (1) tot 3,9 per 100.000 (2). Op een bevolking van 17 miljoen zou dit betekenen dat er 243 tot 663 patiënten in aanmerking komen in Nederland. Officiële getallen in Nederland zijn echter niet bekend, mogelijk is dit aantal een overschatting.

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.