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Fosdenopterin

Nulibry

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
Proprietary name Nulibry
Manufacturer BridgeBio Pharma
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
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
ATMP No
Submission date October 2021
Expected Registration September 2022
Orphan drug Yes
Registration phase Registered
Additional remarks Positieve CHMP opinie ontvangen in juli 2022.

Therapeutic value

Therapeutic value No estimate possible yet
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.
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.
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
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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References AdisInsight

Other information

There is currently no futher information available.