Expand all sections

Apadamtase alfa

TAK-755

Extended indication Congenital (or hereditary) Thrombotic Thrombocytopenic Purpura (cTTP)
Therapeutic value No estimate possible yet
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)
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
ATMP No
Submission date September 2023
Expected Registration August 2024
Orphan drug Yes
Registration phase Positive CHMP opinion

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
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.
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.
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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

Off label use No

Indication extension

Indication extension Yes
Indication extensions Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Other information

There is currently no futher information available.