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The ADZYNMA clinical development programme included a pivotal, randomised, controlled Phase 3 study, the first of its kind in cTTP patients ^{2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596. 3 3. Scully, M, et al. Blood. 2017; 130(19):2055-2063.}

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A Phase 1, first-in-human, dose escalation, safety, and pharmacokinetics study in cTTP patients (NCT02216084) ^{3 3. Scully, M, et al. Blood. 2017; 130(19):2055-2063. 4 4. Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP). Available at: https://www.clinicaltrials.gov/study/NCT02216084. Accessed July 2024.}
 

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A pivotal, randomised, controlled Phase 3 study in cTTP patients (NCT03393975) ^{5 5. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP). Available at: https://clinicaltrials.gov/study/NCT03393975. Accessed July 2024.}
 

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A Phase 3b continuation study in cTTP patients (NCT04683003) ^{6 6. A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura. Available at: https://clinicaltrials.gov/study/NCT04683003. Accessed July 2024.}

ADZYNMA Phase 3 study design ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

A Phase 3, multinational, prospective, open-label, randomised, controlled, two-period crossover trial to evaluate the efficacy and safety of ADZYNMA and standard therapy administered as routine prophylaxis or on-demand treatment in patients with congenital TTP.  ^{2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

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2. Scully M, et al. N Engl J Med. 2024; 390(17):1584-1596.
5. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP). Available at: https://clinicaltrials.gov/study/NCT03393975. Accessed July 2024
7. Takeda Press Release. 5th January 2023. 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/. Accessed July 2024.
8.Takeda Press Release. 26th March 2024. Available at: https://www.takeda.com/newsroom/newsreleases/2024/adzynma-japan-regulatory-approval/. Accessed July 2024./

Study Patient Population ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}  

Patients with severe congenital deficiency of ADAMTS13 (cTTP; defined as plasma ADAMTS13 activity <10%) aged 0–70 years.

Intervention ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

ADZYNMA

Prophylaxis dose regimen:

• 40 IU/kg body weight of ADZYNMA once every other week.

On-demand dose regimen:

• Day 1: Administer 40 IU/kg body weight of ADZYNMA.
• Day 2: Administer 20 IU/kg body weight of ADZYNMA.
• Day 3 and thereafter: 15 IU/kg of body weight once daily until two days after the acute event was resolved.

Comparator ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

Investigator-recommended plasma-based therapies.

Outcomes ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024. 2 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

• The primary outcome measure was the incidence of acute TTP events.*
• Secondary endpoints included the number and incidence of acute cTTP episodes responding to ADZYNMA; time to resolution of clinical symptoms and laboratory parameters; incidence of isolated clinical symptoms (including thrombocytopenia, microangiopathic haemolytic anaemia (MAHA), renal dysfunction, neurological symptoms, and abdominal pain); incidence of adverse events; incidence of antibodies to ADAMTS13; PK properties; health-related quality of life; and resource utilization.
• Exploratory outcome measures included the incidence of subacute (or non-acute) manifestations in patients receiving prophylactic treatment, measured as incidence of composite TTP manifestations (defined as the occurrence of two or more of the following: thrombocytopenia, microangiopathic haemolytic anaemia, renal dysfunction, neurological symptoms or abdominal pain).

*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 microlitre 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 2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.}

ADZYNMA is now an option for cTTP prophylaxis and treatment^{*, 9 9. Mazepa, M. Blood. 2022; 140(7):671-672.}

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

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

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

*Physicians must educate patients and parents on the benefits of prophylaxis to ensure adherence for adequate protection against cTTP relapses.

Acronyms

ADAMTS13, A disintegrin and metalloproteinase with a thrombospondin motifs 13

CHO, Chinese hamster ovary

cTTP, Congenital TTP

DNA, Deoxyribonucleic acid

ERT, Enzyme replacement therapy

IU, International unit 
rADAMTS13
LDH, Lactate dehydrogenase
MAHA, Microangiopathic haemolytic anaemia

PK, Pharmacokinetic

QoL, Quality of life
rADAMTS13, Recombinant ADAMTS13
SmPC, Summary of product characteristics

SoC, Standard of care
TTP, Thrombotic thrombocytopenic purpura
Tx, Treatment

VWF, Von Willebrand factor