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 Registration phase Positive CHMP opinion 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 Unknown 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 Unknown ATMP No Submission date 2025 Expected Registration June 2025 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie april 2025. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration 250 mg References 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 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