Denne sides oplysninger er på engelsk.

Image

PRESCRIBING ADZYNMA

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

*ADZYNMA is a purified bivariant human recombinant A disintegrin and metalloproteinase with thrombospondin motifs 13 (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 dosing1 

Image
Image
Image
Image


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


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


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


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.1

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

 

ADZYNMA indication
 

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.
Image


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 Prescribing Information.

Image


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.

*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

Coming soon

 


ADZYNMA Reconstitution and Administration video

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

ADZYNMA Storage1

Special precautions for storage1

  • 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 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 are no or limited amount of data from 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

 

BREAST-FEEDING

  • There is insufficient information on the excretion of apadamtase alfa in human or animal milk but it is unlikely that it is excreted in human milk due to its high molecular weight.1 The decision either to discontinue nursing 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.
  • 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.
  • 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 20 and 1 paediatric patients aged 2-17 years in the prophylactic and on demand cohorts, respectively. Overall, the safety profile in these paediatric patients was similar to that observed in the adult population. One neonate aged 36 hours old 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 are expected to be the same as in adults.

 

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 elderly patients.

 

RENAL IMPAIRMENT

  • As apadamtase alfa 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.

 

HEPATIC IMPAIRMENT

  • As apadamtase alfa 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.

 

 


Helpful resources

Baxject II Hi-Flow device Troubleshooting Guide

 

Coming soon

ADZYNMA Instructions for Use

Information on how to reconstitute and administer ADZYNMA

Coming soon

Understanding ADZYNMA

A helpful resource that explains how ADZYNMA is given and how it works

Coming soon

.

References and acronyms

  1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. August 2024.
  2. Batchelder L, et al. Blood. 2023;142(Suppl 1):2366.


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, Recombinant ADAMTS13
S/D, Solvent detergent
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura
VWF, Von Willebrand factor

 

Image

Har du brug for mere information?

Kontakt os på medinfoemea@takeda.com.
Oplysninger om produktresuméet kan findes her.
Birvirkninger eller uønskede hændelser skal indberettes til AE.DNK@takeda.com.