Apadamtase alfa Medicine 05 December 2023 Extended indication Congenital (or hereditary) Thrombotic Thrombocytopenic Purpura (cTTP) Therapeutic value No estimate possible yet Total cost Registration phase Positive CHMP opinion Product Active substance Apadamtase alfa Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Congenital (or hereditary) Thrombotic Thrombocytopenic Purpura (cTTP) Current proprietary name Already available biosimilars / generics Proprietary name TAK-755 Manufacturer Takeda Portfolio holder Takeda Mechanism of action Enzyme Route of administration Intravenous Therapeutical formulation Powder for injection / infusion solution Budgetting framework Intermural (MSZ) Centre of expertise https://bloedziekten.nl/ttp/waar-terecht/ Additional remarks TAK-755 replaces the missing or deficient ADAMTS-13 enzyme Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date September 2023 Expected Registration August 2024 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Infusion of plasma or plasma exchange (PEX) to replace ADAMTS-13 is the standard of care in cTTP. Plasma infusions are burdensome for patients and can achieve about 25% of normal ADAMTS-13 levels. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Conceptueel heeft het geneesmiddel meerwaarde. Het werkingsmechanisme is interessant. Er kan een meerwaarde zijn ten opzichte van plasma vanwege de belasting van plasma-infusie/ferese en het bereiken van hogere spiegels, theoretisch is het ook veiliger. Resultaten op overleving en kwaliteit van leven moeten nog afgewacht worden. Het ziet er naar uit dat het minder belastend is. Duration of treatment Frequency of administration 1 times every 2 weeks Dosage per administration 40 IU/kg References NCT03393975; Amsis et al (2022). NEJM. 2022;387:2356-61. DOI: 10.1056/NEJMoa2211113; https://hematologienederland.nl/wp-content/uploads/2021/03/Richtlijn2021-TMA_def-300321.pdf Additional remarks Prophylactic and on-demand treatment with other dosing scheme, monotherapy (without infusion of plasma or plasma exchange). Expected patient volume per year Patient volume 0 - 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expert opinion Additional remarks cTTP is een zeer zeldzaam ziektebeeld. Wereldwijd wordt het aantal patiënten geschat op minder dan 150 patiënten. Het patiëntenaantal in Nederland is niet bekend, maar zal extreem laag zijn. Er wordt verwacht dat er hooguit 5 patiënten in aanmerking zullen komen. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) References Additional remarks Other information Additional remarks