APADAMTASE ALFA WITH CINAXADAMTASE ALFA (RECOMBINANT ADAMTS13)
Information current as at: 1 September 2026
Submission Details
- Brand name:
-
- Adzynma®
- Form and strength:
-
- Powder for injection 500 I.U. with solvent
- Powder for injection 1500 I.U. with solvent
- Submission sponsor:
- TAKEDA PHARMACEUTICALS AUSTRALIA PTY. LTD.
- Condition/indication:
(therapeutic use) -
- Congenital thrombotic thrombocytopenic purpura (cTTP)
- Listing requested:
- To request listing of apadamtase alfa / cinaxadamtase alfa (an enzyme replacement therapy) for the prevention of acute events in adult and paediatric patients with chronic, severe cTTP who have a confirmed ADAMTS13 genetic mutation and ADAMTS13 activity levels less than 10% and who cannot tolerate, or are hypersensitive to, preventative treatment with fresh frozen plasma.
- Funding program:
- PBS Section 100 (Highly Specialised Drugs Program)
- Request authority level:
- Authority Required (Telephone/Online)
- PBAC Submission type:
- New PBS listing (Category 1)
- Comment:
- --
- Other PBAC consideration:
- --
Progress Details
-
Submission received for: - July 2026 PBAC meeting
-
Opportunity for consumer comment: - Open 25/03/2026 and close 20/05/2026 (see PBS Website)
-
PBAC meeting: - Held on 08/07/2026
-
5Lodgement of required documentation:
- 19/08/2026
-
Acceptance of complete documentation:
- Under consideration
-
6Agreement to listing arrangements:
- Has not yet commenced
-
7Government processes:
- Has not yet commenced
-
8Medicine listed on the PBS:
- Has not yet occurred
PBAC Outcome
The PBAC recommended subsidy of recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy through the PBS for both prophylactic and on-demand treatment of patients with chronic, severe congenital thrombotic thrombocytopenia purpura (cTTP).
Enzyme replacement therapy for cTTP works by administering rADAMTS13, which replaces the persons missing ADAMTS13 enzyme which in turn helps restore functions, helping to prevent dangerous blood clots, reduce disease episodes, protect organs, and potentially to improve day-to-day quality of life compared with standard treatment with fresh frozen plasma (FFP).
The PBAC welcomed input from individuals, health care professionals and medical organisations highlighting there is a high need for clinically effective therapies. The PBAC acknowledged the input supported the recommended PBS listing which is broader than the population initially requested by the supplying company.
The PBAC accepted that prophylactic treatment with rADAMTS13 would reduce the risk of breakthrough episodes that may lead to blood clots, low platelet counts, anaemia, and organ damage compared to standard treatment with FFP.
The PBAC was satisfied that in the context of an extremely rare and life-threatening disease with high unmet clinical need, that the cost-effectiveness of rADAMTS13 would be acceptable at the price requested.