Denne sides oplysninger er på engelsk.

Image

SAFETY

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

 

The ADZYNMA safety profile was demonstrated in a Phase 3, open-label, crossover trial (NCT03393975). Adverse events occurred in 71% of the patients with ADZYNMA and in 84% with plasma-based therapy. No neutralising antibodies developed during ADZYNMA treatment.2

Summary of Treatment-Emergent Adverse Events (TEAEs)1,2

Image

 

ADZYNMA prophylaxis resulted in fewer hypersensitivity reactions when compared to SoC2

 

Image
image
image
image
image


In a Phase 3, open-label, crossover trial (NCT03393975), the safety profile of ADZYNMA was demonstrated with adverse events occurring in 71% of the patients with ADZYNMA and in 84% with plasma-based therapy. No neutralising antibodies developed during recombinant ADAMTS13 treatment.2

 

Drug-related TEAEs were 5x more common among patients who received plasma-based therapy vs those who received ADZYNMA (~ 48% vs ~ 9%, respectively).2

 

No ADZYNMA-related serious adverse events were reported.2

 

In Periods 1 & 2, ADZYNMA prophylaxis resulted in 0 hypersensitivity adverse event (n = 45) vs 24 events in 16 patients in the plasma-based therapies group (n = 44).2


ADZYNMA is contraindicated in patients with life-threatening hypersensitivity to ADZYNMA or to any of its excipients.*,1

Hypersensitivity:1

Allergic-type hypersensitivity including anaphylactic reactions may occur. Patients should be informed of the early signs of hypersensitivity reactions including but not limited to tachycardia, tightness of the chest, wheezing and/or acute respiratory distress, hypotension, generalised urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paraesthesia, restlessness, and may progress to anaphylactic shock. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of ADZYNMA and provide appropriate supportive care.

Immunogenicity:1

As with all therapeutic proteins, there is a potential for immunogenicity. Patients may develop antibodies to rADAMTS13 following treatment with ADZYNMA which could potentially result in a decreased response to rADAMTS13. If such antibodies are suspected and there is a lack of efficacy, consider other therapeutic strategies.

*List of excipients. Powder: Sodium chloride, Calcium chloride dihydrate, L Histidine, Mannitol, Sucrose, Polysorbate 80 (E433). Solvent: Water for injections.


Adverse reactions in the ADZYNMA clinical trials1

In a Phase 3, open label, crossover trial (NCT03393975), no ADZYNMA related serious adverse events were reported.2
 

The safety profile of ADZYNMA was evaluated in two studies:1

  1. A global Phase 3, prospective, randomised, controlled, open-label, multicentre, two-period crossover study followed by a single-arm continuation period
  2. A long-term continuation study for patients who completed the Phase 3 study\

 

The most common adverse reactions reported in clinical studies were headache (31.5%), diarrhoea (17.8%), dizziness (16.4%), upper respiratory tract infection (15.1%), nausea (13.7%), and migraine (11%).1
 

MEDRA SYSTEM ORGAN CLASS (SOC) ADVERSE REACTION BY PREFERRED TERM (PT) FREQUENCY CATEGORY BY SUBJECT*

Infections and Infestations

Upper respiratory tract infection

Very common

Blood and Lymphatic System Disorders

Thrombocytosis

Common

Nervous System Disorders

Headache

Migraine

Dizziness

Somnolence

Very common

Very common

Very common

Common

Gastrointestinal Disorders

Diarrhoea

Nausea

Constipation

Abdominal distension

Very common

Very common

Common

Common

General Disorders and Administration Site Conditions

Asthenia

Feeling hot

Common

Common

Investigations

ADAMTS13 activity abnormal

Common
Image



No patients receiving ADZYNMA had adverse events leading to treatment discontinuation or interruption.2


* Adverse reactions are listed above by MedDRA system organ class and by frequency. Frequencies are defined as follows: very common (≥ 1/10); common (≥ 1/100 to < 1/10); uncommon (≥ 1/1 000 to < 1/100); rare (≥ 1/10 000 to < 1/1 000); very rare (< 1/10 000); not known (cannot be estimated from the available data). Within each System Organ Class (SOC), ADRs are presented in order of decreasing frequency. Within each frequency grouping, ADRs are presented in order of decreasing seriousness.


 

References and acronyms

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

ADAMTS13, A disintegrin and metalloproteinase with a thrombospondin motifs 13
ADR, Adverse drug reaction
cTTP, Congenital TTP
CHO, Chinese hamster ovary
DNA, Deoxyribonucleic acid
ERT, Enzyme replacement therapy
IP, Investigational product
PT, Preferred term
rADAMTS13, Recombinant ADAMTS13
SmPC, Summary of product characteristics
SoC, Standard of care
SOC, System organ class
TEAE, Treatment-emergent adverse event
TTP, Thrombotic thrombocytopenic purpura

 

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.