Brand Name

Adzynma

Generic Name
Apadamtase
View Brand Information
FDA approval date: November 09, 2023
Form: Kit

What is Adzynma (Apadamtase)?

ADZYNMA is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” indicated for prophylactic or on demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura [see Use in Specific Populations.

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Related Clinical Trials

Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
Enrollment Status: Recruiting
Publish Date: October 07, 2025
Intervention Type: Drug

Summary: This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from recombin...

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Related Latest Advances

Apadamtase Alfa: First Approval.
Apadamtase Alfa: First Approval.
Journal: Drugs
Published: February 28, 2024
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Brand Information

ADZYNMA (Apadamtase Alfa)
1INDICATIONS 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)
2DOSAGE AND ADMINISTRATION
For intravenous use after reconstitution only.
2.1Dosage
  • Each vial of ADZYNMA is labeled with the actual rADAMTS13 activity, measured in terms of its potency in International Units (IU).
  • Calculate administration dose and volume based on the patient's body weight using the actual potency (and not the nominal potency) as printed on ADZYNMA vial.
  • For Intravenous (IV) Infusion at a rate of 2 to 4 mL per minute.
Prophylactic Therapy
The 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
A guide for dosing ADZYNMA for on demand treatment of an acute event is provided in
2.2Preparation and Administration
  • Use aseptic technique (clean and germ-free) throughout the procedure.
  • Check the expiration date of the product prior to use.
  • Do not use ADZYNMA if the expiration date has passed.
Reconstitution
  1. Prepare a clean, germ-free, flat surface and gather all the materials you will need for the reconstitution and infusion.
  1. Do not use ADZYNMA if the expiration date has passed.
  2. Use ADZYNMA
  3. Allow the vials of ADZYNMA and diluent to reach room temperature before use.
  4. Wash and dry your hands thoroughly, and put on clean exam gloves.
  5. Remove plastic caps from the ADZYNMA and diluent vials and place the vials on a flat surface (
  1. Wipe the rubber stoppers with an alcohol swab and allow them to dry prior to use (
  1. Open the BAXJECT II Hi-Flow device package by peeling away the lid, without touching the inside (
  1. Turn the package with the BAXJECT II Hi-Flow device upside down and place it over the top of the diluent vial. Press straight down until the
  1. Grip the BAXJECT II Hi-Flow device package at its edge and pull the package off the device (
  1. Turn the system over so that the diluent vial is now on top. Press the BAXJECT II Hi-Flow device straight down until the purple plastic spike pierces through the ADZYNMA powder vial stopper (Figure G). The vacuum will draw the diluent into the ADZYNMA powder vial.
  2. You may notice some bubbles or foam – this is normal and should soon disappear. Wait until foam or bubbles dissipate before administration.
  • Swirl the connected vials
  1. Visually inspect the reconstituted solution for particulate matter before administration. The solution should be clear and colorless in appearance.
  2. If the dose requires more than one vial of ADZYNMA, repeat step 1 to step 12 to reconstitute each vial.
  1. Take off the
  1. Turn the system upside down (ADZYNMA vial is now on top). Draw the reconstituted solution into the syringe by pulling the plunger back slowly (Figure K).
  2. Use ADZYNMA
  3. Do not administer ADZYNMA in the same tubing or container at the same time with other medicinal products for infusion.
  1. If a patient is to receive more than one vial of ADZYNMA, the contents of multiple vials can be drawn into the same syringe. Repeat this process for all reconstituted vials of ADZYNMA until the total volume to be administered is reached.
  2. Disconnect the syringe and attach a suitable injection needle or an infusion set.
  3. Point the needle up and remove any air bubbles by gently tapping the syringe with your finger and slowly and carefully pushing air out of the syringe and needle.
  4. Apply a tourniquet and clean the chosen infusion site with an alcohol swab (
  1. Insert the needle into the vein and remove the tourniquet.
  2. Infuse the reconstituted ADZYNMA
  1. Take the needle out of the vein, place a cotton ball or gauze on the infusion site, and apply pressure for several minutes to stop bleeding.
  2. Place the needle, syringe, and empty vials in a puncture-resistant sharps container.
3DOSAGE FORMS AND STRENGTHS
ADZYNMA is a lyophilized powder in single-dose vials containing nominally 500 or 1500 International Units (IU) per vial and comes with 5 mL of Sterile Water for Injection. Each carton and vial label for ADZYNMA states the actual potency of ADAMTS13 in IU.
4CONTRAINDICATIONS
ADZYNMA is contraindicated in patients who have manifested life threatening hypersensitivity reactions to ADZYNMA or its components
5ADVERSE 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.
5.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety profile of ADZYNMA was evaluated in one, prospective, randomized, active-controlled, open-label, multicenter, two-period crossover study (Study 1). The adverse drug reactions (ADR) are listed in
6DESCRIPTION
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). ADZYNMA is produced and formulated without the addition of any exogenous raw materials of human or animal origin in the cell culture, purification, or formulation of the final product. The purification process for rADAMTS13 does not include use of a monoclonal antibody reagent. To enhance viral safety, the production process also incorporates two dedicated viral clearance steps – a solvent/detergent treatment step for inactivation and a 20 nm filtration step for removal of viruses. Recombinant ADAMTS13 has a molecular weight of approximately 172 kDa. Proteins that may be present in the final product, other than rADAMTS13, are trace quantities of host cell (CHO) proteins.
ADZYNMA (rADAMTS13) is a sterile, nonpyrogenic, preservative free, white powder supplied in single-dose vials for IV use after reconstitution. Each single-dose vial contains nominally 500 IU or 1500 IU of rADAMTS13, sodium chloride (9.4 mg), calcium chloride dihydrate (1.6 mg), L-histidine (16.7 mg), mannitol (161.4 mg), sucrose (53.8 mg), and polysorbate 80 (2.7 mg). Each vial of ADZYNMA is labeled with the specific number of units of ADAMTS13 potency expressed in IU as measured with a fluorescence resonance energy transfer (FRET) assay using a synthetic 73-amino-acid peptide (FRETS-VWF73). The potency assignment employs an ADAMTS13 concentrate standard that is referenced to a WHO (World Health Organization) international standard for ADAMTS13 concentrates and is evaluated by appropriate methodology to ensure accuracy of the results.
After reconstitution with 5 mL of Sterile Water for Injection, USP, the 500 IU and the 1500 IU vials result in a nominal potency of 100 IU/mL and 300 IU/mL, respectively. All dosage strengths yield a clear, colorless solution, free from particles with a pH of approximately 7.0.
7CLINICAL STUDIES
ADZYNMA was studied in a global, prospective, randomized, active-controlled, open-label, multicenter, two-period crossover study followed by a single arm continuation period (Study 1) evaluating the efficacy and safety of the prophylactic and on demand ERT with ADZYNMA compared to plasma-based therapies in patients with cTTP.
8REFERENCES
  1. Asmis, L. M., Serra, A., Krafft, A., Licht, A., Leisinger, E., Henschkowski-Serra, J., et al. 2022. Recombinant ADAMTS13 for Hereditary Thrombotic Thrombocytopenic Purpura. N Engl J Med, 387, 2356-2361.
  2. Alwan, F., C. Vendramin, R. Liesner, A. Clark, W. Lester, T. Dutt, W. Thomas, R. Gooding, T. Biss, H. G. Watson, N. Cooper, R. Rayment, T. Cranfield, J. J. van Veen, Q. A. Hill, S. Davis, J. Motwani, N. Bhatnagar, N. Priddee, M. David, M. P. Crowley, J. Alamelu, H. Lyall, J. P. Westwood, M. Thomas and M. Scully (2019). “Characterization and treatment of congenital thrombotic thrombocytopenic purpura.” Blood 133(15): 1644-1651.
  3. Sukumar, S., B. Lämmle and S. R. Cataland (2021). “Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management.” J Clin Med 10(3): 536.
9HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
ADZYNMA (rADAMTS13) is a sterile, nonpyrogenic, preservative free, white powder supplied in single-dose vials packaged with:
  • one vial with 5 mL sterile water for injection, USP
  • one BAXJECT II Hi-FLOW needleless transfer device
  • one syringe
  • one 25 gauge infusion set
  • two individually packaged alcohol swabs
  • one package insert
ADZYNMA (rADAMTS13) is available in the following strengths:
The actual ADAMTS13 potency in international units is printed on the label of each ADZYNMA vial and carton.
Components are not made with natural rubber latex.
Storage and Handling
  • Store at refrigerated temperature 2°C to 8°C (36°F to 46°F) for up to 36 months from the date of manufacture until expiration date stated on the ADZYNMA vial label and carton. Within this period, ADZYNMA may be stored at room temperature not to exceed 30°C/86°F for a period up to 6 months. After storage at room temperature, do not return to the refrigerator.
  • Do not use beyond the expiration date printed on the ADZYNMA vial label or carton or if not stored properly.
  • Do not freeze.
  • Store in the original box and protect from extreme exposure to light.
  • Use reconstituted product immediately or within 3 hours after reconstitution when stored at room temperature.
  • Do not use if the solution in the syringe is cloudy or contains particles.
  • Discard any unused reconstituted product after 3 hours.
10PATIENT COUNSELING INFORMATION
Advise the patient:
  • To read the FDA-approved patient labeling (
  • About early signs of hypersensitivity reactions, including tachycardia, tightness of the chest, wheezing and/or acute respiratory distress, hypotension, generalized urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paresthesia, and restlessness. Advise patients to discontinue use of the product if these symptoms occur and seek immediate emergency treatment with appropriate supportive care.
  • To consult with their physicians or healthcare provider prior to traveling. While traveling, advise patients to bring an adequate supply of ADZYNMA based on their current regimen of treatment.
11PRINCIPAL DISPLAY PANEL - 500 IU Kit Carton
NDC 64764-140-05
Rx Only
ADZYNMA
ADAMTS13, recombinant-krhn
For Intravenous Use After Reconstitution Only
500 IU
Single-dose vial
Dosage: See prescribing information. Prior to administration of ADZYNMA,
read the instructions for use.
Storage: Store refrigerated at 36°F to 46°F (2°C to 8°C) until the expiration date
stated on the carton, do not freeze. Can be stored at room temperature not to
exceed 86°F/30°C for a period of 6 months, do not return to refrigerator.
Store in the original carton to protect from light. See prescribing information.
Reconstitution: Use 5 mL Sterile Water for Injection, USP. Gently swirl, do not shake.
Do not refrigerate after reconstitution. Use within 3 hours of reconstitution.
Discard unused portion.
Keep out of the reach of children.
Date removed from
PRINCIPAL DISPLAY PANEL - 500 IU Kit Carton
12PRINCIPAL DISPLAY PANEL - 1500 IU Kit Carton
NDC 64764-145-05
Rx Only
ADZYNMA
ADAMTS13, recombinant-krhn
For Intravenous Use After Reconstitution Only
1500 IU
Single-dose vial
Dosage: See prescribing information. Prior to administration of ADZYNMA,
read the instructions for use.
Storage: Store refrigerated at 36°F to 46°F (2°C to 8°C) until the expiration date
stated on the carton, do not freeze. Can be stored at room temperature not to
exceed 86°F/30°C for a period of 6 months, do not return to refrigerator.
Store in the original carton to protect from light. See prescribing information.
Reconstitution: Use 5 mL Sterile Water for Injection, USP. Gently swirl, do not shake.
Do not refrigerate after reconstitution. Use within 3 hours of reconstitution.
Discard unused portion.
Keep out of the reach of children.
Date removed from
PRINCIPAL DISPLAY PANEL - 1500 IU Kit Carton