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ADZYNMA is an enzyme replacement therapy (ERT) indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA can be used for all age groups. ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}  

• ADZYNMA is intended for intravenous use after reconstitution only. ADZYNMA 500 IU and ADZYNMA 1500 IU powder and solvent for solution for injection should be administered at a rate of 2 to 4 mL per minute.
 ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}
• Recommended prophylactic dose is 40 IU/kg of body weight 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 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}  

• For on-demand enzyme replacement therapy: In case of acute thrombotic thrombocytopenic purpura (TTP) episode, the recommended dose of ADZYNMA to treat acute TTP episodes is 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.
 ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}
• In clinical studies, single doses up to 160 IU/kg were used and their safety profile was generally consistent with results from clinical study results in cTTP patients. In case of overdose, based on the pharmacological action of apadamtase alfa, there is the potential for increased risk of bleeding.

For full dosage and administration instructions, see Summary of Product Characteristics. ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

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Home or self-administration under the supervision of a healthcare professional may be considered for patients who are tolerating their injections well^{*, 1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

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 2. Batchelder L, et al. Blood. 2023;142(Suppl 1):2366.}

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 2. Batchelder L, et al. Blood. 2023;142(Suppl 1):2366.}

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

*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 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

ADZYNMA Reconstitution and Administration video
 

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

ADZYNMA Storage ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

Special precautions for storage ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

• Store in a refrigerator (2–8 °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 ADZYNMA is removed from refrigeration.
• Shelf life: 3 years.

Storage conditions after reconstitution of the medicinal product ^{1 1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.}

• 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.

Acronyms

ADAMTS13, A disintegrin and metalloproteinase with a thrombospondin motifs 13

cTTP, Congenital TTP

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

rADAMTS13, Recombinant ADAMTS13
S/D, Solvent detergent
SmPC
SmPC, Summary of product characteristics

TTP, Thrombotic thrombocytopenic purpura
VWF, Von Willebrand factor