Eplontersen Medicine 06 June 2023 Extended indication Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Eplontersen Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Neurological disorders other Extended indication Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. Current proprietary name Already available biosimilars / generics Proprietary name Wainzua Manufacturer AstraZeneca Portfolio holder AstraZeneca Mechanism of action Antisense oligonucleotide Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Prealbumin expression inhibitor; RNA interference. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date October 2023 Expected Registration March 2025 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie oktober 2024. Therapeutic value Current treatment options Huidige behandelopties zijn: diflunisal en tafamidis. Daarnaast zal het middel moeten gaan concurreren met patisiran, inotersen en vutrisiran. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Eplontersen toonde effectiviteit aan in de fase 3 trial voor hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) na 66 weken. n=166, persbericht: er werd een relevante verandering gezien in de 'modified Neuropathy Impairment Score'. De concurrentiepositie van eplontersen ten opzichte van vutrisiran is echter lastig, omdat vutrisiran veel minder vaak toegepast dient te worden dan eplontersen. Duration of treatment Average 0 continuous Frequency of administration 1 times a month Dosage per administration 45mg References NCT04136184; Expertopinie Additional remarks Vergelijkende studie met inotersen (toediening eens per week). Expected patient volume per year Patient volume 30 - 60 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Er zijn volgens een persbericht van AstraZeneca 40.000 patiënten met ATTRv-PN wereldwijd. Op dit moment ontvangen er 29 patiënten patisiran. De verwachting is dat er mogelijk 30 tot 60 patiënten in aanmerking zullen gaan komen. Expected cost per patient per year Cost References Additional remarks lijst prijs is nog niet bekend. 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 Yes Indication extensions ATTR Cardiomyopathy (CM) References Fabrikant Additional remarks Deze indicatie wordt in 2027 verwacht. Other information Additional remarks