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ADZYNMA is the first and only FDA-approved recombinant ADAMTS13 therapy for patients with congenital thrombotic thrombocytopenic purpura (cTTP) designed to address its root cause.1*

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*ADAMTS13 enzyme deficiency.2,3

In a PK analysis of a clinical study, ADZYNMA was shown to increase the levels of ADAMTS13.1

ADZYNMA is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” ADAMTS13 (rADAMTS13) indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

ADZYNMA was studied in a global prospective, randomized, active-controlled, open-label, two-period crossover study followed by a single-arm continuation period (Study 1) evaluating the efficacy and safety of the prophylactic and on-demand enzyme replacement therapy with ADZYNMA compared to plasma-based therapies in patients with cTTP. In the prophylactic efficacy and safety evaluation, patients received ADZYNMA 40 IU/kg weekly or every other week or plasma-based therapy for 6 months (Period 1), then crossed over to the other treatment for 6 months (Period 2). All patients received ADZYNMA for the single arm continuation period (Period 3).1 cTTP is an ultra-rare disease with ADAMTS13 genetic mutations leading to ADAMTS13 deficiency.2,3

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Mechanism of Action
& Pharmacokinetics

ADZYNMA is a recombinant form of the endogenous ADAMTS13.

Compared to plasma-based therapies:

  • ADZYNMA 40 IU/kg IV administration resulted in approximately 4- to 5-fold higher ADAMTS13 activity exposures and lower inter-subject variability.
  • Mean time duration above 10% ADAMTS13 activity and mean average activity§ were both approximately 3- to 4-fold higher.

Cmax, AUC

Pharmacokinetic Parameters of ADAMTS13 Activity following IV Administration of ADZYNMA in cTTP Patients ≥12 Years Old1
Mean (SD)
Min; Max
1.15 (0.25)
0.78; 1.56
57.6 (13.9)
37.0; 88.2
AUC(0-168 h)​
57.2 (13.67)
36.2; 87.2
Cave(0-168 h)​
0.30 (0.07)
0.20; 0.46
Duration ADAMTS13
Activity above 10% (days)
5.8 (1.2)
4.5; 8.9

AUC = area under ADAMTS13 activity-time curve; Cave (0-168 h) = average
ADAMTS13 activity from 0 to 168 hours dosing interval; Cmax =  maximum ADAMTS13 activity.

Note: 1 IU/mL ADAMTS13 activity corresponds to 100% average normal activity.

SD = standard deviation.

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In Periods 1 and 2:

No acutea or subacutec TTP events were reported in patients receiving ADZYNMA (n=37). In patients receiving plasma-based therapies (n=38), one acute TTP event occurred in a patient and five subacute events were reported in four patients.

Mean annualized event rate of thrombocytopeniad was 2.0 in patients receiving ADZYNMA as compared to 4.44 in patients receiving plasma-based therapies.

Mean annualized event rate of microangiopathic hemolytic anemiad was 0.38 in patients receiving ADZYNMA as compared to 1.47 in patients receiving plasma-based therapies.

Prophylactic Cohort Efficacy Results in cTTP Patients ≥12 Years of Age (Periods 1 and 2)
Acute TTP eventsa
Number of subjects with event 0 1
Mean annualized event rate (SD)b 0 (0.000) 0.05 (0.304)
Subacute TTP eventsc
Number of subjects with event 0 4
Mean annualized event rate (SD)b 0 (0.000) 0.27 (0.836)
TTP manifestationsd
Thrombocytopenia eventse
Number of subjects with event 9 19
Mean annualized event rate (SD)b 2.0 (4.706) 4.44 (6.312)
Microangiopathic hemolytic anemia eventsf
Number of subjects with event 5 11
Mean annualized event rate (SD)b 0.38 (1.028) 1.47 (3.274)

SD = standard deviation; TTP = thrombotic thrombocytopenic purpura

bAnnualized event rate for a subject = number of events/duration of the observation period (years). Data shown are non‑model based.

dTTP manifestations not shown also included renal dysfunction, neurological symptoms and abdominal pain. The clinical significance of the observed difference for the presented manifestations is unknown.

For definitions of Acute and Subacute events, as well as Thrombocytopenia and Microangiopathic Hemolytic Anemia events, please click here click here

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The safety profile of ADZYNMA was evaluated in Study 1.

Adverse Reactions Reported in >5% of Patients Treated with ADZYNMA1
Adverse Reaction (N=48)
n (%)a
Headache 15 (31.3)
Diarrhea 8 (16.7)
Migraine 7 (14.6)
Abdominal pain 6 (12.5)
Nausea 6 (12.5)
Upper respiratory tract infection 6 (12.5)
Dizziness 5 (10.4)
Vomiting 5 (10.4)

N = Total number of patients treated with ADZYNMA in Study 1.

n = Number of patients who had at least one event in the category.

aPercentages by patient were calculated using the number of all subjects who had the listed adverse event.

Prophylactic Therapy

Recommended prophylactic dosage regimen of ADZYNMA is as follows:​​

  • Administer 40 IU/kg body weight once every other week​
  • The prophylactic dosing frequency may be adjusted to 40 IU/kg body weight once weekly based on prior prophylactic dosing regimen or clinical response ​

On Demand Therapy

For on-demand treatment of an acute event:

Treatment Day 1​ Treatment Day 2​ Treatment Day 3 and Beyond
40 IU/kg 20 IU/kg 15 IU/kg once daily until two days after the acute event is resolved.
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ADZYNMA is a lyophilized powder in single-dose vials containing nominally 500 or 1500 International Units (IU) per vial and comes with 5 mL of Sterile Water for Injection.

Infusing ADZYNMA 40 IU/kg would take approximately 2.5-5 minutes for a 70 kg patient (rate 2 to 4 mL per minute) utilizing the 1500 IU vials.

aAcute TTP events were defined in protocol by a drop in platelet count (≥50% of baseline or a platelet count <100,000/µL) and an elevation of lactate dehydrogenase (LDH) (>2× baseline or >2× upper limit normal (ULN)).

cSubacute events were defined by a thrombocytopenia event or a microangiopathic hemolytic anemia event; and organ‑specific signs and symptoms including but not limited to renal dysfunction events, neurological symptoms events, fever, fatigue/lethargy, and/or abdominal pain.

eThrombocytopenia events were defined as a drop in platelet count ≥25% of baseline or a platelet count <150,000/µL.

fMicroangiopathic hemolytic anemia events were defined as an elevation of LDH >1.5×baseline or >1.5 ×ULN.

ADZYNMA (ADAMTS13, recombinant-krhn) Indication & Important Safety Information


ADZYNMA is contraindicated in patients who have experienced life-threatening hypersensitivity reactions to ADZYNMA or its components.

Hypersensitivity Reactions: Allergic-type hypersensitivity, including anaphylactic reactions, may occur with ADZYNMA. Patients should be educated about early signs of hypersensitivity such as tachycardia, chest tightness, wheezing and/or acute respiratory distress, hypotension, generalized urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paresthesia, and restlessness. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of ADZYNMA and provide appropriate supportive care.

Immunogenicity: There is a potential for immunogenicity with ADZYNMA. Patients may develop neutralizing antibodies to ADAMTS13, which could potentially result in a decreased or lack of response to ADAMTS13. Patients may develop antibodies to host cell proteins which could potentially result in adverse reactions. There are no data on immunogenicity with ADZYNMA or to host cell proteins in previously untreated patients (subjects naïve to plasma-based products).

Adverse Reactions: The most commonly observed adverse reactions (>5% of subjects) associated with ADZYNMA are headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.

Use in Specific Populations: The safety of ADZYNMA for use during pregnancy has not been established in controlled clinical trials. Limited data are insufficient to inform a drug associated risk of adverse developmental outcomes. There is no information regarding the presence of ADZYNMA in human milk, its effects on milk production, or the breastfed infant.

To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceuticals U.S.A., Inc. at 1-877-TAKEDA-7 (1-877-825-3327) or FDA at 1-800-FDA-1088 or

Please see full Prescribing Information, including information for patients.