Alpha-1 antitrypsin Medicine 09 January 2018 Extended indication Alpha-1 protease inhibitor monotherapy for treatment of Alpha-1 Antitrypsin deficiency associated Emphysema pulmonary in adults and elderly. Therapeutic value No estimate possible yet Total cost € 82,600,000.00 Registration phase Clinical trials Product Active substance Alpha-1 antitrypsin Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Lung diseases other Extended indication Alpha-1 protease inhibitor monotherapy for treatment of Alpha-1 Antitrypsin deficiency associated Emphysema pulmonary in adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Glassia Manufacturer Grifols Portfolio holder Grifols Mechanism of action Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Kamada heeft de registratieaanvraag teruggetrokken in juni 2017. De EMA heeft destijds aangegeven dat de aanvraag niet voldoende onderbouwd is voor toelating. Inmiddels heeft er registratie bij de FDA plaatsgevonden. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date October 2026 Expected Registration December 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Suppletietherapie Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a week Dosage per administration 60mg/kg of 120mg/kg References NCT01983241 (SPARTA) Additional remarks Expected patient volume per year Patient volume 700 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Longfonds (1) Additional remarks Alpha-1 is een zeldzame ziekte. Bij ongeveer 700 Nederlanders is de diagnose gesteld, maar waarschijnlijk lopen er veel mensen met alpha-1 rond die niet weten dat zij de ziekte hebben. De schatting is dat tussen de 5.000 en 10.000 Nederlanders lijden aan alpha-1 (1). Expected cost per patient per year Cost € < 118,000.00 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 Drugs.com (1); Kamada Ltd. Annual report on Form 20-F for the fiscal year ended Dec 31, 2023. SEC; 2024 (2). Additional remarks GLASSIA is een vloeibaar medicijn dat wekelijks via een intraveneus infuus wordt toegediend. Elke flacon bevat 1.000mg alfa-1-PI in 50ml oplossing. De aanbevolen dosering is 60mg per kilogram lichaamsgewicht (1). Voor iemand van 80 kilo komt dit neer op 4.800mg, oftewel vijf flacons per behandeling. De kosten voor 10mg GLASSIA bedragen $5,353 (2), oftewel $535,30 per flacon van 1.000mg. De kosten bedragen dan $2.676,50 per week, wat neerkomt op circa $139.000 of €118.000 per jaar. Potential total cost per year Total cost € 82,600,000.00 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