Apadamtase alfa, sold under the brand name Adzynma, is an enzyme replacement therapy used to treat thrombotic thrombocytopenic purpura. It is a human recombinant a disintegrin and metalloproteinase with thrombospondin motifs 13, and it is given by injection into a vein. The most common side effects are headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting. It was approved for medical use in the United States in November 2023 and in the European Union in August 2024.
Facts
Drug ClassRecombinant ADAMTS13 enzyme replacement therapy 2 Mechanism of ActionRecombinant form of the plasma zinc metalloprotease ADAMTS13, which cleaves large and ultra-large von Willebrand factor multimers into smaller units, reducing the platelet binding properties of VWF and its propensity to form microthrombi 2 Approved UseProphylactic or on demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) 2 First Approved / Isolated Year Significant RiskNeutralizing antibodies to ADAMTS13 with serious outcomes including death have occurred following administration 2 Classification
Approval Status Route of Administration Connections
Long-Form Articles
Treats
Source Apadamtase alfa (Wikipedia)
Sources
1. Apadamtase alfa (Wikipedia)
Lead section
Apadamtase alfa was approved for medical use in the United States in November 2023, and in the European Union in August 2024.
Lead section [route-of-administration]
It is given by injection into a vein.
Treats: Thrombotic thrombocytopenic purpura, Lead section [treats]
used for the treatment of thrombotic thrombocytopenic purpura
View the Source2. ADZYNMA prescribing information (DailyMed, FDA-approved label)
12.1 Mechanism of Action
by cleaving large and ultra-large VWF multimers to smaller units and thereby reducing the platelet binding properties of VWF
12.1 Mechanism of Action, first sentence
ADZYNMA is a recombinant form of the endogenous ADAMTS13.
1 Indications and Usage
ADZYNMA is indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients
5.1 Neutralizing Antibodies
Neutralizing antibodies to ADAMTS13 with serious outcomes including death have occurred following ADZYNMA administration
View the Source3. ChEMBL: ChEMBL molecule record for Apadamtase alfa (CHEMBL4594607)
ChEMBLmolecule_typeQuote, molecule_type
"molecule_type": "Enzyme"
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