TransCon PTH (palopegteriparatide)

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 remarks	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 remarks	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 remarks	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.

Patient volume

< 50

Market share is generally not included unless otherwise stated.

References

Inschatting NatPar HS

Additional remarks

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 remarks	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.

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.