Nonacog beta pegol Medicine 06 June 2023 Extended indication Extension of indication to include treatment and prophylaxis of bleeding in children below 12 years of age with haemophilia B including previously untreated patients for REFIXIA. Therapeutic value Possible equal value Total cost Registration phase Registration application pending Product Active substance Nonacog beta pegol Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Hemostasis promoting medication Extended indication Extension of indication to include treatment and prophylaxis of bleeding in children below 12 years of age with haemophilia B including previously untreated patients for REFIXIA. Current proprietary name Already available biosimilars / generics Proprietary name Refixia Manufacturer Novo Nordisk Portfolio holder Mechanism of action Unknown Route of administration Unknown Therapeutical formulation Powder for injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date December 2022 Expected Registration October 2023 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Er zijn diverse factor IX stollingsfactor preparaten beschikbaar voor kinderen <12 jaar (Benefix, Rixubis, Alprolix, Idelvion). Therapeutic value Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation De PARADIGM-6 trial in kinderen (inclusief PUPs) met hemofilie B <2% laat goede effectiviteit zien van dit langwerkende FIX product. Er is meerwaarde ten opzichte van de bestaande kortwerkende FIX preparaten. Er is waarschijnlijk gelijke effectiviteit ten opzichte van de andere 2 langwerkende FIX preparaten. Refixia (Nonacog beta pegol) is tot nu toe het enige preparaat dat alleen voor > 12 jaar geregisterd is. Duration of treatment Frequency of administration Dosage per administration References NCT01467427 Additional remarks Expected patient volume per year Patient volume < 45 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Erfelijkheid.nl Additional remarks Het totaal aantal hemofilie B patiënten wordt geschat op 400 in Nederland. Hemofilie B komt hoofdzakelijk bij mannen voor. Er zijn ongeveer 2 miljoen kinderen in Nederland in de leeftijd tot 12 jaar. Dit betekent dat er ongeveer 45 patiënten in deze leeftijdsklasse vallen. Mogelijk zal een gedeelte van deze groep gebruik gaan maken van de behandeling. 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