Diflunisal Medicine 03 June 2025 Extended indication Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. Therapeutic value No estimate possible yet Total cost € 5,040,000.00 Registration phase Registered Product Active substance Diflunisal Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Neurological disorders other Extended indication Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. Current proprietary name Already available biosimilars / generics Proprietary name Attrogy Manufacturer Purpose Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date 2025 Expected Registration June 2025 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie april 2025. Therapeutic value Current treatment options Tafamidis 20mg Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Op dit moment wordt diflunisal al voorgeschreven in Nederland door middel van een artsenverklaring. De therapeutische waarde is vergelijkbaar met de positie van tafamidis 20mg wat betreft effectiviteit in stadium 1 & 2, maar de bijwerkingen zijn wel een nadeel. Tafamidis heeft alleen voor stadium 1 een registratie. Duration of treatment Frequency of administration Dosage per administration 250 mg References NCT00294671 Additional remarks Expected patient volume per year Patient volume 45 - 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Richtlijn diagnostiek en behandeling van erfelijke ATTR amyloïdose GRaCE (1); Expertopinie expertisecentrum (2); Schmidt et al. Muscle & Nerve. 2018 (3). Additional remarks hATTR amyloïdose is uiterst zeldzaam. De geschatte incidentie in Nederland is 3 per jaar (100 patiënten in 33 jaar, van 1985 tot 2018), de geschatte prevalentie 3 per miljoen (nu circa 50 onder controle op 17 miljoen mensen) (2). De literatuur onderbouwt een prevalentie van 45 patiënten (3). De ziekte treft evenveel mannen als vrouwen. De prevalentie in Nederland wordt geschat op 50 patiënten (1). Expected cost per patient per year Cost € 105,000.00 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 Gipdatabank Additional remarks Er is niets bekend over de prijs per gebruiker van diflunisal. De vergoeding van tafamidis was in 2024 per gebruiker €104.990. Potential total cost per year Total cost € 5,040,000.00 Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks