Extended indication Hypoparathyroidism in adults
Therapeutic value No estimate possible yet
Total cost 5,000,000.00
Registration phase Clinical trials

Product

Active substance TransCon PTH (palopegteriparatide)
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Hypoparathyroidism in adults
Manufacturer Ascendis
Mechanism of action Receptor antagonist
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Additional comments Long-acting prodrug of parathyroid hormone (PTH). In its prodrug inactive form, PTH(1-34) is transiently bound to the TransCon technology which releases unmodified teriparatide, via non-enzymatic hydrolysis of the TransCon linker.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date December 2022
Expected Registration 2023
Orphan drug Yes
Registration phase Clinical trials
Additional comments Weesgeneesmiddel voor de volgende indicaties: Zeldzame hypoparathyreoïdie Genetische hypoparathyreoïdie Familiale geïsoleerde hypoparathyreoïdie

Therapeutic value

Current treatment options NatPar, calcium met vitamine D.
Therapeutic value No estimate possible yet
Frequency of administration 1 times a day
Dosage per administration 18 mcg
References PaTHway

Expected patient volume per year

Patient volume

< 50

Market share is generally not included unless otherwise stated.

References Inschatting NatPar HS
Additional comments Patiënten met hypoparathyreoidie maar met calcium neerslagen zoals bij hypercalciurische complicaties zoals nefrocalcinose, calcium neerslagen in de huid et cetera. Dit vormt een beperkt deel van de hypoparathyreoidiepopulatie. De inschatting is dat dit ongeveer 50 patiënten zal bedragen. In 2021 waren er 13 patiënten die NatPar kregen.

Expected cost per patient per year

Cost 100,000.00
References Expert opinie;
Additional comments De prijs zal waarschijnlijk rond de €100.000 per patiënt per jaar liggen.

Potential total cost per year

Total cost

5,000,000.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

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.