ADZYNMA (ADAMTS13, recombinant-krhn)

ADZYNMA (ADAMTS13, recombinant-krhn)

Medical Administration – intravenous

Diagnosis considered for coverage:

Congenital Thrombotic Thrombocytopenic Purpura (cTTP): Indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

Coverage Criteria:

For diagnosis of thrombotic thrombocytopenic purpura (cTTP):

• Diagnosis of congenital thrombotic thrombocytopenic purpura (cTTP), AND
• Molecular genetic testing confirms mutations in the ADAMTS13 gene, AND
• Trial and inadequate response, contraindication or intolerance to plasma-based infusions

Reauthorization Criteria:

For diagnosis of thrombotic thrombocytopenic purpura (cTTP):

• Patient demonstrates positive clinical response to therapy, AND
• Trial and inadequate response, contraindication or intolerance to plasma-based infusions

Coverage Duration: 

• Initial: 12 months
• Reauthorization: 24 months

Authorization is not covered for the following:
The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.
 

Additional Information: 

• Dosing 
  • Prophylactic Therapy
    • 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
  • 
• Additional Information
  • Acute TTP events were defined in protocol by a drop in platelet count (≥50% of baseline or a platelet count <100,000/µL) and an elevation of lactate dehydrogenase (LDH) (>2× baseline or >2× upper limit normal (ULN)). Subacute events were defined by a thrombocytopenia event or a microangiopathic hemolytic anemia event; and organ specific signs and symptoms including but not limited to renal dysfunction events, neurological symptoms events, fever, fatigue/lethargy, and/or abdominal pain. Thrombocytopenia events were defined as a drop in platelet count ≥25% of baseline or a platelet count <150,000/µL. Microangiopathic hemolytic anemia events were defined as an elevation of LDH >1.5×baseline or >1.5 ×ULN. 
  • A trial of plasma-based infusions would be acceptable for both prophylactic and acute use in cTTP. Adzynma may be an option for those patients who have risk of antibody production, allergic reactions, time-constraints (few hours with plasma-based infusions), refractory to plasma-based infusions, volume restrictions (e.g., renal failure) etc.

Policy Updates:

• 6/1/2024 (policy effective date)- New Iwilfin Criteria (P&T 5/20/2024) (P&T Meeting May)

References:

1. Iwilfin Prescribing Information. US WorldMeds, Louisville, KY, December 2023. 
2. PDQ® Pediatric Treatment Editorial Board. PDQ Neuroblastoma Treatment. Bethesda, MD: National Cancer Institute. Updated <02/13/2024>. Available at: https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq. Accessed <2/28/2024>. [PMID: 26389190] 
3. Consult with pediatric hematology/oncology consultant. 26 Feb 2024