Glibenclamide Medicine 09 January 2018 Extended indication Amglidia is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children. Therapeutic value No judgement Total cost Registration phase Registered and reimbursed Product Active substance Glibenclamide Domain Metabolism and Endocrinology Reason of inclusion Main indication Diabetes Extended indication Amglidia is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children. Current proprietary name Already available biosimilars / generics Proprietary name Amglidia Manufacturer Ammtek Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Dit middel wordt al langer toegepast bij diabetes, maar betreft bij neonatale diabetes een weesgeneesmiddel. In veel pasgeboren baby's met neonatale diabetes, produceren de cellen in de pancreas wel insuline maar zijn de cellen niet in staat om het vrij te geven in het bloed. Het gebrek aan insuline in het bloed veroorzaakt de diabetes symptomen. De verwachting is dat Glibenclamide een effect heeft op de KATP kanalen waardoor de mogelijkheid van cellen om insuline in het bloed vrij te geven herstellen. Er wordt ook gedacht dat Glibenclamide de rest van het lichaam effectiever laat reageren op insuline. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine ATMP Unknown Submission date November 2016 Expected Registration May 2018 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie februari 2018. Geregistreerd in mei 2018. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 17 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks EMA Orphan designation: 0.01 in 10,000 people in the European Union (EU). Dit betekent 17 per 17 miljoen inwoners. Expected cost per patient per year Cost References Additional remarks 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