Fosdenopterin Medicine 07 December 2021 Extended indication Metabolic disease molybdenum cofactor deficiency (MoCD) Type A Therapeutic value No estimate possible yet Total cost € 1,560,000.00 Registration phase Registered Product Active substance Fosdenopterin Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Metabolic disease molybdenum cofactor deficiency (MoCD) Type A Current proprietary name Already available biosimilars / generics Proprietary name Nulibry Manufacturer BridgeBio Pharma Portfolio holder Mechanism of action Other Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks A synthetic version of cPMP, Molybdenum cofactor replacements. Fabrikant: BridgeBio Pharma / Origin Biosciences Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date October 2021 Expected Registration September 2022 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen in juli 2022. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het effect van de eerste studie lijkt groot te zijn. Patiënten die met fosdenopterin behandeld werden hadden een overleving van 84% na drie jaar, tegenover 55% van de niet behandelde patiënten. Duration of treatment Frequency of administration 1 times a day Dosage per administration 0,9 mg/kg References https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214018s000lbl.pdf, NCT02629393 Additional remarks Titration Schedule Preterm Neonates (Gestational Age Less than 37 weeks): Initial Dosage= 0.4mg/kg once daily, Month 1= 0.7mg/kg once daily, Month 3= 0.9mg/kg once daily Term Neonates (Gestational Age 37 weeks and Above): Initial Dosage= 0.55mg/kg once daily, Month 1= 0.75mg/kg once daily, Month 3= 0.9mg/kg once daily For patients one year of age or older, the recommended dosage of NULIBRY is 0.9mg/kg (based on actual body weight) administered as an intravenous infusion once daily. Expected patient volume per year Patient volume 2 - 4 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Erfelijkheid.nl, Orphanet Additional remarks Over de hele wereld zijn er meer dan 100 mensen met molybdeen co-factor deficiëntie. De incidentie is 1:200.000 geboortes. Maximaal 1 nieuwe patiënt per jaar. Na 10 jaar is 20% van de onbehandelde patiënten nog in leven. In Nederland zijn nu 4 patiënten bekend, waarvan er reeds 2 worden behandeld. Expected cost per patient per year Cost € > 520,000.00 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 drugs.com Additional remarks Prijs in de Verenigde Staten betreft $1,452 per 9,5mg poeder voor intraveneuze injectie. Potential total cost per year Total cost € 1,560,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References AdisInsight Additional remarks Other information Additional remarks