Dasiglucagon Medicine 04 June 2024 Extended indication Infants and children aged 7 days to 12 years with congenital hyperinsulinism (CHI). Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Dasiglucagon Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Infants and children aged 7 days to 12 years with congenital hyperinsulinism (CHI). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Zealand Portfolio holder Mechanism of action Glucagon analogue Route of administration Subcutaneous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise 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) Type of trajectory Unknown Particularity New therapeutical formulation ATMP No Submission date 2025 Expected Registration 2026 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Diazoxide Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 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 10µg/h, in addition to standard-of-care therapy. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 Cost References 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 Total cost 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