Encaleret Medicine 05 December 2023 Extended indication Autosomal Dominant Hypocalcemia Type 1 (ADH1). Therapeutic value No estimate possible yet Total cost 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). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer BridgeBio Pharma Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Calcium-sensing receptor antagonist; Osteogenesis stimulant; Parathyroid hormone receptor agonist. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date March 2026 Expected Registration April 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. 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. Duration of treatment Frequency of administration Dosage per administration References NCT05680818 (CALIBRATE) Additional remarks Expected patient volume per year Patient volume 257 - 702 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 18 miljoen zou dit betekenen dat er 257 tot 702 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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks