Caplacizumab Medicine 09 January 2018 Extended indication Acquired thrombotic thrombocytopenic purpura (aTTP) - adjunctive therapy Therapeutic value Possible added value Total cost € 2,805,000.00 Registration phase Registration application pending Product Active substance Caplacizumab Domain Cardiovascular diseases Reason of inclusion Main indication Other non-oncological hematological medications Extended indication Acquired thrombotic thrombocytopenic purpura (aTTP) - adjunctive therapy Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Ablynx Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Caplacizumab bindt met vWF en verhindert op die manier de vorming van microscopische bloedklonters. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New medicine ATMP Unknown Submission date March 2017 Expected Registration August 2018 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Plasmaferese en immuunsuppressie Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Toegevoegd aan bestaande behandeling geeft het snellere remissie van trombotische trombocytopenische purpura en tevens een reductie van trombotische complicaties. Duration of treatment Average 40 day / days Frequency of administration 1 times a day Dosage per administration 10 mg References Peyvandi et al.; N Engl J Med 2016; 374:511-522 Additional remarks Expected patient volume per year Patient volume 15 - 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Uitgaande van een incidentie in Nederland van 0,37 per 100.000 kan het aantal nieuwe volwassen (ouder dan 18 jaar) TTP patiënten in Nederland geschat worden op max. 49 patiënten per jaar. Het aantal patiënten dat momenteel niet reageert op de eerstelijnsbehandeling of een recidief krijgt wordt rond de 30% geschat (15 patiënten per jaar). Expected cost per patient per year Cost € 85,000.00 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 detijd.be Additional remarks Detijd.be: 'Ablynx mikt op een prijs van $175.000 per behandeling in de VS en €85.000 in Europa.' Het zal gebruikt worden in aanvulling op huidige behandeling. Potential total cost per year Total cost € 2,805,000.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks