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Dasiglucagon

Extended indication Infants and children aged 7 days to 12 years with congenital hyperinsulinism (CHI).
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Dasiglucagon
Domain Metabolism and Endocrinology
Reason of inclusion Indication extension
Main indication Metabolic diseases
Extended indication Infants and children aged 7 days to 12 years with congenital hyperinsulinism (CHI).
Manufacturer Zealand
Mechanism of action Glucagon analogue
Route of administration Subcutaneous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Medical experts also indicated that shifts in patient management might be required to implement effective use of dasiglucagon as it is administered as a continuous subcutaneous infusion via a pump device. The developer has partnered with Deka Research and Development Corp (Manchester, New Hampshire, USA) to develop a wearable subcutaneous infusion pump to deliver dasiglucagon.

Registration

Registration route Centralised (EMA)
Particularity New therapeutical formulation
ATMP No
Submission date 2024
Expected Registration 2025
Registration phase Clinical trials

Therapeutic value

Current treatment options Diazoxide
Therapeutic value No estimate possible yet
Dosage per administration 10 µg/h
References NCT04172441
Additional remarks In phase 3 clinical trials, patients receive dasiglucagon via a continuous subcutaneous infusion pump starting at a dose rate of 10 µg/h, in addition to standard-of-care therapy.

Expected patient volume per year

References Orphanet (1); NTVG(2);
Additional remarks De wereldwijde gemiddelde prevalentie bij de geboorte bedraagt 1 op 27.000, maar kan tot 1 op 2.500 bedragen in gemeenschappen met substantiële bloedverwantschap. In Aalten en Silvolde werd een hoge prevalentie waargenomen (1 op 6.930 inwoners), waarschijnlijk door een gemeenschappelijke voorouder (2).

Expected cost per patient per year

Additional remarks De prijs van deze toedieningsvorm zal mogelijk hoger uitvallen dan de huidige toedieningsvorm. De fabrikant zal mogelijk een hoger bedrag vragen aangezien het om een zeldzaam ziektebeeld gaat.

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.