Overview
11 DESCRIPTION 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).
Mechanism of Action
12 CLINICAL PHARMACOLOGY 12.1 Mechanism of Action ADZYNMA is a recombinant form of the endogenous ADAMTS13. ADAMTS13 is a plasma zinc metalloprotease that regulates the activity of von Willebrand factor (VWF) by cleaving large and ultra-large VWF multimers to smaller units and thereby reducing the platelet binding properties of VWF and its propensity to form microthrombi. 2, 3 12.2 Pharmacodynamics VWF antigen (VWF:AG) and VWF:ristocetin cofactor activity (VWF:RCo) were used to assess VWF platelet binding activity. Following IV doses of ADZYNMA at the recommended dose, both VWF:AG and VWF:RCo transiently decreased for 1 to 2 days with a 15% to 25% change from baseline. 12.3 Pharmacokinetics The PK profile of ADZYNMA was determined based on clinical trial ADAMTS13 activity data analyses.
Indications
- 1 INDICATIONS AND USAGE ADZYNMA (ADAMTS13, recombinant-krhn) is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13) indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) [see Use in Specific Populations (8.4) , Clinical Studies (14) ]. ADZYNMA (ADAMTS13, recombinant-krhn) is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13) indicated for prophylactic or on demand enzyme replacement...
Common Doses
- 4 mL
Dosage
2 DOSAGE AND ADMINISTRATION For intravenous use after reconstitution only. For intravenous use after reconstitution only. ( 2 ) Prophylactic Therapy Administer 40 IU/kg body weight once every other week intravenously at a rate of 2 to 4 mL per minute. ( 2.1 ) The prophylaxis dosing frequency may be adjusted to 40 IU/kg body weight once weekly based on prior prophylactic dosing regimen or clinical response. ( 2.1 ) On-Demand Therapy Administer intravenously at a rate of 2 to 4 mL per minute: 40 IU/kg body weight on day 1. ( 2.1 ) 20 IU/kg body weight on day 2. ( 2.
Contraindications
- 4 CONTRAINDICATIONS ADZYNMA is contraindicated in patients who have manifested life threatening hypersensitivity reactions to ADZYNMA or its components [see Description (11) ] . Do not use in patients who have manifested life threatening hypersensitivity reactions to ADZYNMA or its components. ( 4 )
Side Effects
- 6 ADVERSE REACTIONS Most common adverse reactions (>5% of subjects) reported in clinical trials were headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting. Most common adverse reactions (incidence >5%) are headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting. ( 6.1 ) 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 www.fda.gov/medwatch . 6.
Interactions
- No major interactions listed on the FDA label. Always consult a healthcare professional.