banner

Clinical Development Program overview

ADZYNMA▼ is an enzyme replacement therapy (ERT) indicated for the treatment of “A disintegrin and metalloproteinase with thrombospondin motifs 13” (ADAMTS13) deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA can be used for all age groups.1

*ADZYNMA is a purified bivariant human recombinant ADAMTS13 ,(rADAMTS13) expressed in Chinese Hamster Ovary (CHO) cells using recombinant DNA technology (a mixture of native rADAMTS13 Q23 and variant rADAMTS13 R23 with a controlled range of the two variants ratio), referred to as rADAMTS13.

The ADZYNMA clinical development program included a pivotal, open-label, randomised, controlled, two-period crossover Phase 3 Study, the first-of-its-kind in patients with cTTP. 2,3

A Phase 1, first-in-human, dose escalation, safety, and pharmacokinetics study in patients with cTTP ( NCTO2216084) 4

A pivotal, randomised, controlled Phase 3 study in patients with cTTP (NCT03393975)5

A phase 3b continuation study in patients with cTTP (NCT04683003)6

ADZYNMA PHASE 3 Study design 1,2

A phase 3, multinational, prospective, open-label, randomised, controlled, two-period crossover trial (NCTO3393975) evaluated the efficacy and safety of ADZYNMA and plasma-based therapy administered as routine prophylaxis or on-demand treatment in patients with congenital thrombotic thrombocytopenic purpura (cTTP). 2

 

graph

Adapted from Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.

A Phase 3 study aimed to assess the safety and efficacy of ADZYNMA in the prevention and treatment of acute episodes of thrombotic thrombocytopenic purpura (TTP) in patients with congenital TTP. (NCT03393975). 2,5

The study evaluated the clinical benefit of ADZYNMA across efficacy, pharmacokinetic, safety, and immunogenicity endpoints in patients with severe cTTP. 5

Patients could be enrolled to prophylaxis treatment and were randomised one-to-one in a Phase 3, open-label, crossover trial (NCT03393975) to receive either ADZYNMA followed by plasma-based therapies or plasmabased therapies followed by ADZYNMA.2

In the Phase 3, open-label, crossover trial (NCT03393975), in addition to the cohort receiving prophylactic treatment, a cohort included patients entering the trial to control an acute event with on-demand treatment. When patients experienced an event, they were randomised one-to-one to receive either ADZYNMA or plasma-based therapies until the acute TTP event was resolved. After resolution, patients could opt to join the prophylactic cohort.2

STUDY PATIENT POPULATION 1,2

Patients with severe congenital deficiency of ADAMTS13 (cTTP; defined as plasma ADAMTS13 activity <10%) ages 0-70

INTERVENTION 1

ADZYNMA

Prophylaxis enzyme replacement therapy: 

  • 40 IU/kg of body weight once every other week.
  • The prophylaxis dosing frequency may be adjusted to 40 UI/kg of body weight once weekly bases on clinical response.

In case of acute thrombotic thrombocytopenic purpura (TTP) event, the recommended dose of ADZYNMA TO TREAT ACUTE TTP events is as follows: 

  • 40 IU/kg of body weight on Day 1
  • 20 IU/kg of body weight on Day 2
  • 15 IU/kg of body weight starting Day 3 once daily until two days after the acute event is resolved.

 

COMPARATOR 1,2

Investigator-recommended plasma-based therapies

OUTCOMES  2,5,8
  • Primary endpoint: ADZYNMA prophylaxis for a median of 13,3 months was associated with zero acute thrombotic thrombocytopenic purpura (TTP) events in a phase 3, open-label, crossover trial among patients with congenital TTP (NCT03393975).*
  • Secondary endpoints included the number and incidence of acute cTTP events responding to ADZYNMA

Adverse events occurred in 71% of the patients with ADZYNMA and in 84% with plasma-derived therapy in periods 1 and 2 of the trial.2

*An acute TTP event was defined as a decrease in the platelet count by at least 50% from baseline or to less than 100,000 per µL and an elevation of the lactate dehydrogenase (LDH) level to more than 2 times the baseline value or more than 2 times the upper limit of the normal range (ULN).2,5

ADZYNMA is now an option for congenital thrombotic thrombocytopenic purpura (cTTP) prophylaxis and treatment. Physicians must educate patients and caregivers on the benefits of prophylaxis to ensure adherence for adequate protection against both stroke and cTTP relapses 9

 

Takeda is committed to advancing treatment options in rare genetics and haematology

This is a step forward in the management of cTTP and brings new possibilities to patients

Takeda is committed to advancing disease management, including treatment options and improving QoL for those living with cTTP

References and acronyms

  1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. Last approved version.
  2. Scully M, et al. N Engl J Med. 2024; 390(17):1584-1596
  3. Takeda Press Release. 5th January 2023. Takeda Announces Favorable Phase 3 Safety and Efficacy Results of TAK-755 as Compared to Standard of Care in Congenital Thrombotic Thrombocytopenic Purpura (cTTP). Accessed February 2025. Available at: https://www.takeda.com/newsroom/newsreleases/2022/takeda-announces-favorable-phase-3-safety-and-efficacy-results-of-tak-755-as-compared-to-standard-of-care-in-congenital-thrombotic-thrombocytopenic-purpura-cttp/
  4. Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP). NCT02216084. Updated 5th May 2021. Accessed February 2025. https://clinicaltrials.gov/study/NCT02216084
  5. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP): NCT03393975. Updated 4th June 2024. Accessed February 2025. https://clinicaltrials.gov/study/NCT03393975
  6. A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura. NCT04683003. 29th April 2024. Accessed February 2025. https://clinicaltrials.gov/study/NCT04683003
  7. Takeda Press Release. 26th March 2024. Takeda Announces Approval of ADZYNMA® Intravenous Injection 1500 (apadamtase alfa / cinaxadamtase alfa) in Japan for Patients with Congenital Thrombotic Thrombocytopenic Purpura (cTTP). Accessed February 2025. Available at: https://www.takeda.com/newsroom/newsreleases/2024/adzynma-japan-regulatory-approval/
  8. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596. Supplementary appendix
  9. Mazepa M. Blood. 2022;140(7):671–672.

 

ADAMTS13, A disintegrin and metalloproteinase with thrombospondin motifs 13

CHO, Chinese hamster ovary
cTTP, Congenital thrombotic thrombocytopenic purpura

DNA, Deoxyribonucleic acid

ERT, Enzyme replacement therapy

EU, European Union

H, Half

IU, International unit

LDH, Lactate dehydrogenase

MAHA, Microangiopathic haemolytic anaemia

PBT, Plasma-based therapy

Ph, Phase

PK, Pharmacokinetic

QoL, Quality of life

rADAMTS13, Recombinant ADAMTS13

SmPC, Summary of product characteristics

TTP, Thrombotic thrombocytopenic purpura

Tx, Treatment

ULN, Upper limit of normal

US, United States

 

smpc

price

 

Image


Need further information?

Contact Medical Information

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 of the SmPC for how to report adverse reactions.

C-APROM/BE/ADZ/0003 November 2025