Sirolimus Medicine 12 June 2018 Extended indication Rapamune is indicated for the treatment of patients with sporadic lymphangioleiomyomatosis with moderate lung disease or declining lung function Therapeutic value No judgement Total cost € 200,000.00 Registration phase Registered and reimbursed Product Active substance Sirolimus Domain Lung diseases Reason of inclusion Indication extension IND Main indication Lung other Extended indication Rapamune is indicated for the treatment of patients with sporadic lymphangioleiomyomatosis with moderate lung disease or declining lung function Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks mTOR remmer Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration August 2018 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks FDA heeft sirolimus voor indicatie LAM op 28 mei 2015 goedgekeurd. Sectie Interstitiele Longziekten (SIL) van de NVALT is van mening dat sirolimus geindiceerd is bij lymfangioleiomyomatose (LAM) met abnormale (FEV1 <70%pred) of dalende longfunctie; in de loop van 2018 verschijnt hierover een zogenaamd 'Standpunt LAM'. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Internationale richtlijnen en standpunt LAM NVALT. Duration of treatment Frequency of administration Dosage per administration 1-2 mg/dag References McCormack et al. American Journal of Respiratory and Critical Care Medicine 2016; 194(6): September 15; Gupta et al. American Journal of Respiratory and Critical Care Medicine 2017 Nov 15;196(10):1337-1348; McCormack et al. N Engl J Med 2011;364(17):1595-606; Ando et al. Respiratory Investigation 2013;51:175-83.; Taveira-DaSilva et al. Ann Intern Med 2011;154(12):797-805; Neurohr et al. Respir Res 2011;12:66.; Barrera P et al. AnnalsATS 2013;10(4): 408-409.; Moua et al. Am J Respir Crit Care Med 2012;186(4):389-90.; Ohara et al. Ann Thorac Surg 2008;86:e7-8. Additional remarks Expected patient volume per year Patient volume < 80 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Struik et al. Expert Opinion on Orphan Drugs 2015;3(4):393-401. Additional remarks Expected cost per patient per year Cost € 2,500.00 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 Medicijnkosten.nl, GIP-databank. Additional remarks Sirolimus tablet 1 mg € 4,44 en 2 mg € 8,66. Kosten per patiënt per jaar €2.441,00 in 2016 voor een andere indicatie; profylaxe van orgaanafstoting na niertransplantatie. Potential total cost per year Total cost € 200,000.00 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