Apadamtase alfa

ADZYNMA is indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). 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).

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.

For intravenous use after reconstitution only. Prophylactic Therapy Administer 40 IU/kg body weight once every other week intravenously at a rate of 2 to 4 mL per minute. The prophylaxis 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 Administer intravenously at a rate of 2 to 4 mL per minute: 40 IU/kg body weight on day 1. 20 IU/kg body weight on day 2. 15 IU/kg body weight on day 3 and beyond until two days after the acute event is resolved.

ADZYNMA is contraindicated in patients who have manifested life-threatening hypersensitivity reactions to ADZYNMA or its components. Do not use in patients who have manifested life-threatening hypersensitivity reactions to ADZYNMA or its components.

Boxed warning (neutralizing antibodies to adamts13): Neutralizing antibodies to ADAMTS13 with serious outcomes, including death, have been reported in patients treated with ADZYNMA. Neutralizing antibodies may result in decreased or lack of response to recombinant or plasma-derived ADAMTS13. Monitor all patients closely for ADAMTS13 activity and development of ADAMTS13 neutralizing antibodies. Neutralizing antibodies to ADAMTS13 with serious outcomes including death have been reported in patients with cTTP following administration of ADZYNMA. Monitor all patients, including those who were previously exposed or naïve to plasma-based products, by assessing ADAMTS13 activity and ADAMTS13 neutralizing antibodies prior to initiation of ADZYNMA and periodically during the course of treatment. Hypersensitivity reactions may occur.

Most common adverse reactions (incidence >5%) are headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting.

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 freeze. Do not use beyond the expiration date printed on the ADZYNMA vial label or carton or if not stored properly. 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.