Palopegteriparatide Medicine 07 December 2022 Extended indication Hypoparathyroidism in adults Therapeutic value Possible added value for a subgroup Total cost € 5,000,000.00 Registration phase Registered Product Active substance Palopegteriparatide Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Hypoparathyroidism in adults Current proprietary name Already available biosimilars / generics Proprietary name Yorvipath Manufacturer Ascendis Portfolio holder Mechanism of action Receptor antagonist Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise 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) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date December 2022 Expected Registration November 2023 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in september 2023. Therapeutic value Current treatment options NatPar, calcium met vitamine D. Therapeutic value Possible added value for a subgroup This assessment does not indicate any potential inclusion in the package. Substantiation De meeste mensen zullen behandeld worden met vitamine D en calcium. Echter zijn er ook patiënten die opgenomen worden vanwege een hypercalciëmie. Voor deze patiënten zal dit geneesmiddel mogelijk een uitkomst zijn. Duration of treatment Frequency of administration 1 times a day Dosage per administration 18 mcg References PaTHway Additional remarks Expected patient volume per year Patient volume < 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 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. 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 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks