PRESCRIBING

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 (PTTc). 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 rADAMTS13.¹

ADZYNMA prophylactic dosing¹

ADZYNMA is intended for intravenous use after reconstitution only. ADZYNMA 500 IU and ADZYNMA 1,500 IU powder and solvent for solution for injection should be administered at a rate of 2 to 4 mL per minute.¹

The prophylactic dose of ADZYNMA is 40 IU per kg of body weight, administered once every other week.   The prophylaxis dosing frequency may be adjusted to 40 IU/kg of body weight once weekly based on clinical response.¹

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 and 15 IU/kg of body weight starting Day 3 once daily until two days after the acute event is resolved (see table below).^{^1}

In clinical studies, single doses up to 160 IU/kg were used and their safety profile was generally consistent with results from clinical trial results in cTTP patients. In case of overdose, based on the pharmacological action of rADAMTS13, there is the potential for increased risk of bleeding.¹

For full dosage and administration instructions, see Summary of Product Characteristics.¹

On demand dosing

DAY	1	2	3 and beyond until two days after the acute event is resolved
DOSAGE	40 IU/kg	20 IU/kg	15 IU/kg

Important dosage and administration instructions:¹

For intravenous use after reconstitution only.
The dose is calculated based on patient body weight.

For full dosage and administration instructions, see Summary of Product Characteristics.¹

Home or self administration under the supervision of a healthcare professional may be considered for patients who are tolerating their injections well.^1,*

In a cross-sectional cohort study with 307 patients (including 25 with cTTP), survey responses indicated that 137 patients (44.6%) would prefer to receive treatment at home; 125 patients (40.7%) would prefer either a doctor or a nurse to administer their treatment at home.²

A cross-sectional cohort study in patients with cTTP, identified that a high proportion (60.9%) were willing to trade 12 months of their lives to receive treatment at home. Additionally, 59.0% and 56.4% of patients, respectively, were willing to trade 12 months to ensure that complications did not necessitate treatment in a hospital setting and that travelling to a hospital/clinic was not required to receive prophylaxis.²

For further information about ADZYNMA home or self-administration, please refer to the Summary of Product Characteristics.¹

*The decision to have a patient move to home or self-administration should be made after evaluation and recommendation by the treating physician. Appropriate training should be given by the treating physician and/or nurse to the patient and/or caregiver prior to initiation of home or self‑administration. Dose and administration rate should remain constant while at home, and not be changed without consulting the treating physician. If the patient experiences early signs of hypersensitivity during the home administration, the administration process should be stopped immediately, and appropriate treatment should be initiated. Subsequent injections need to occur in a clinical setting. Treatment should be closely followed by the treating physician.¹

Download the ADZYNMA self-administration guide for further information

ADZYNMA Reconstitution and Administration video

See exactly how ADZYNMA is reconstituted and administered by watching this short video.

ADZYNMA Storage and Handling¹

Special precautions for storage¹

Store in a refrigerator (2°C 8°C).
Do not freeze.
Store in the original package in order to protect from light.
ADZYNMA may be stored at room temperature up to 30°C for a period of up to 6 months in lyophilized form, but not exceeding the expiry date.
Do not return ADZYNMA to refrigerated storage after storage at room temperature.
Record on the carton the date and time ADZYNMA is removed from refrigeration.
The shelf life for an unopened vial of ADZYNMA is 3 years.

Storage conditions after reconstitution of the medicinal product¹

Chemical and physical in use stability has been demonstrated for 6 hours at 25°C.
From a microbiological point of view, unless the method of opening/ reconstituting/dilution precludes the risks of microbial contamination, the product should be used immediately. If not used immediately, in use storage times and conditions are the responsibility of the user.

Use of ADZYNMA in special populations¹

Besides body-weight dosing regimen, no intrinsic factors such as age, gender, race, baseline estimated glomerular filtration rate (eGFR), and baseline bilirubin were identified as covariates impacting ADAMTS13 pharmacokinetics.¹

PREGNANCY

There is a limited amount of data on the use of ADZYNMA in pregnant women.¹
Animal studies do not indicate direct or indirect harmful effects with respect to reproductive toxicity.¹
The use of ADZYNMA during pregnancy may only be considered after a thorough individual risk benefit analysis by the treating physician before and during treatment.¹

BREASTFEEDING

There is insufficient data on the excretion of rADAMTS13 in human or animal milk but it is unlikely that it is excreted in human milk due to its high molecular weight.¹The decision either to discontinue breastfeeding or discontinue ADZYNMA should take into account the importance of this medicinal product to the mother.¹

PAEDIATRIC POPULATION

The recommended body weight-based dosing regimen in paediatric patients is the same as in adults.¹
Based on the results from population pharmacokinetics analysis, it may be more likely for infants <10 kg body weight to require adjustment to dosing frequency from every other week to once weekly dosing.¹
There is limited information from controlled studies of ADZYNMA in paediatric patients. The safety assessment in paediatric patients is based on the safety data from one Phase 3 Clinical Study comparing ADZYNMA to plasma-based therapies (fresh frozen plasma [FFP], pooled solvent/detergent [S/D] treated plasma, or factor VIII: von Willebrand factor [FVIII-VWF] concentrates, as assigned by the investigator) and one Phase 3b Study. The studies included paediatric patients aged 2-17 years in the prophylactic (n=20) and on-demand (n=1) cohorts. Overall, the safety profile in these paediatric patients was similar to that observed in the adult population. One 36-hour-old neonate was treated with ADZYNMA in a compassionate use program and had no reported safety or immunogenicity concerns after 2 years of prophylactic treatment. Frequency, type, and severity of adverse reactions in children treated with ADZYNMA are expected to be the same as in adults.¹

ELDERLY

There is limited data on the use of ADZYNMA in patients over 65 years of age. Based on the results from population pharmacokinetics analysis, no dose adjustment is required for eldery patients.¹

RENAL IMPAIRMENT

As rADAMTS13 is a recombinant protein with a high molecular weight, it is not excreted renally and no dose adjustment is needed for patients with renal impairment.¹

HEPATIC IMPAIRMENT

As rADAMTS13 is a recombinant protein with high molecular weight, it is cleared via catabolism (rather than hepatic metabolism), and no dose adjustment is needed for patients with hepatic impairment.¹

References and acronyms

ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. Last approved version.
Batchelder L, et al. Blood. 2023;142(Suppl 1):2366-2368. Abstract 2366.

ADAMTS13, A disintegrin and metalloproteinase with thrombospondin motifs 13
cTTP, Congenital thrombotic thrombocytopenic purpura
CHO, Chinese hamster ovary
DNA, Deoxyribonucleic acid
eGFR, Estimated glomerular filtration rate
ERT, Enzyme replacement therapy
FFP, Fresh frozen plasma
FVIII, Factor VIII
IU, International unit
rADAMTS13, Recombinant ADAMTS13
S/D, Solvent detergent
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura
VWF, von Willebrand factor

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▼ 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/0006 November 2025

PRESCRIBING

ADZYNMA prophylactic dosing1