Liposomaal amikacine Medicine 09 January 2018 Extended indication Longinfecties door Mycobacterium avium complex, niet reagerend op eerdere therapie. Therapeutic value Possible added value Total cost € 1,120,000.00 Registration phase Clinical trials Product Active substance Liposomaal amikacine Domain Infectious diseases Reason of inclusion New medicine (specialité) Main indication Bacterial infections Extended indication Longinfecties door Mycobacterium avium complex, niet reagerend op eerdere therapie. Current proprietary name Already available biosimilars / generics Proprietary name ALIS Manufacturer Insmed Portfolio holder Mechanism of action Antibiotic Route of administration Inhalation Therapeutical formulation Liquid for inhalation vapor Budgetting framework Extramural (GVS) Centre of expertise Radboudumc, afd. Longziekten, Nijmegen. Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New therapeutical formulation ATMP Unknown Submission date 2019 Expected Registration 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Amikacine (onder de merknaam Arikayce) is eerder teruggetrokken tijdens de EMA registratieprocedure door Insmed op 8 juni 2016. De data waarop de aanvraag was gebaseerd werd ontoereikend geacht. Huidige fase III resultaten lijken veelbelovend. Therapeutic value Current treatment options Rifampicine (of rifabutin) en ethambutol en macrolide antibioticum (claritromycine danwel azitromycine). Bij ernstige infecties wordt intraveneuze amikacine toegevoegd. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 6 month / months Frequency of administration 1 times a day Dosage per administration 590 mg References Fabrikant; Olivier et al. Am J Respir Crit Care Med. 2017 Mar 15;195(6):814-823. Additional remarks Expected patient volume per year Patient volume 20 - 35 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References RIVM Additional remarks In Nederland zijn er ongeveer 700 gevallen nontuberculeuze myobacterieinfecties per jaar (2009). Indien de indicatie voor therapie-naieve Mycobacterium avium complex longinfecties eerder wordt verkregen zal dit een verdubbeling van het aantal in aanmerking komende patiënten opleveren. Expected cost per patient per year Cost € 30,000.00 - 50,000.00 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 bron: SPS Additional remarks Mogelijk meerkosten. Prijs is nog niet vastgesteld. In Amerika al wel een prijs: "approved in US where it will be priced it at $363 per vial, or $132,495 annually if taken daily as directed". Verwachting in Nederland eenmalig €30.000-€50.000 per patiënt, o.b.v. 6 maanden behandeling (expert-opinie). Potential total cost per year Total cost € 1,120,000.00 Total cost for sluice Additional remarks Off label use Off label use Yes Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Therapie-naïeve Mycobacterium avium complex longinfecties. Mogelijk in 2020. References Additional remarks Other information Additional remarks