Extended indication Metabolic disease molybdenum cofactor deficiency (MoCD) Type A
Therapeutic value No estimate possible yet
Registration phase Clinical trials

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 (US)
Manufacturer BridgeBio Pharma
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments A synthetic version of cPMP, Molybdenum cofactor replacements. Fabrikant: BridgeBio Pharma / Origin Biosciences

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2022
Expected Registration 2023
Orphan drug Yes
Registration phase Clinical trials
Additional comments Door FDA al geregistreerd

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 comments Titration Schedule Preterm Neonates (Gestational Age Less than 37 weeks): Initial Dosage= 0.4 mg/kg once daily, Month 1= 0.7 mg/kg once daily, Month 3= 0.9 mg/kg once daily Term Neonates (Gestational Age 37 weeks and Above): Initial Dosage= 0.55 mg/kg once daily, Month 1= 0.75 mg/kg once daily, Month 3= 0.9 mg/kg once daily For patients one year of age or older, the recommended dosage of NULIBRY is 0.9 mg/kg (based on actual body weight) administered as an intravenous infusion once daily.

Expected patient volume per year

Patient volume

< 10

Market share is generally not included unless otherwise stated.

References Erfelijkheid.nl, Orphanet
Additional comments 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. Naar schatting zijn dit zo'n 6 patiënten in Nederland, maximaal 10.

Expected cost per patient per year

References drugs.com
Additional comments Prijs in Amerika betreft $1439,63 per 9,5mg poeder voor intraveneuze injectie.

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

Indication extension No
References AdisInsight

Other information

There is currently no futher information available.