Pharmacy Prior Authorization Criteria

Adzynma (ADAMTS13, recombinant-krhn)

Indications for Prior Authorization

Adzynma (ADAMTS13, recombinant-krhn)

• For diagnosis of 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).

Criteria

Adzynma

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• 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 [B, 11]

Adzynma

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Patient demonstrates positive clinical response to therapy
AND
• Trial and inadequate response, contraindication or intolerance to plasma-based infusions [B, 11]

P & T Revisions

2024-02-12

References

1. Adzynma Prescribing Information. Takeda Pharmaceuticals USA, Inc. Lexington, MA. November 2023.
2. Manufacturer drug website. Available at: https://www.adzynma.com/. Accessed February 6, 2024.
3. ClinicalTrials.gov. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP). Available at: https://classic.clinicaltrials.gov/ct2/show/NCT03393975?cond=nct03393975&draw=2&rank=1. Accessed January 6, 2024.
4. ClinicalTrials.gov. A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura. Available at: https://classic.clinicaltrials.gov/ct2/show/NCT04683003?cond=nct04683003&draw=2&rank=1. Accessed January 6, 2024.
5. Sukumar, S., Lammle, B., Cataland, S. Thrombotic Thrombocytopenia Purpura: Pathophysiology, Diagnosis, and Management. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7867179/. Accessed January 6, 2024.
6. Alwan, F., Vendramin, C., Liesner, R., et al. Characterization and treatment of congenital thrombotic thrombocytopenic purpura. Available at: https://www.sciencedirect.com/science/article/pii/S0006497120426357?via%3Dihub. Accessed January 6, 2024.
7. Asmis, L., Serra, A., Krafft, A., et al. Recombinant ADAMTS13 for Hereditary Thrombotic Thrombocytopenic Purpura. Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2211113. Accessed January 6, 2024.
8. UptoDate: Hereditary thrombotic thrombocytopenic purpura (hTTP). Available at: https://www.uptodate.com/contents/hereditary-thrombotic-thrombocytopenic-purpura-http?search=cttp&source=search_result&selectedTitle=1~37&usage_type=default&display_rank=1. Accessed January 6, 2024.
9. Zheng X., Vesely, S., Cataland, S., et al. ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. Available at: https://onlinelibrary.wiley.com/doi/epdf/10.1111/jth.15010. Accessed January 6, 2024.
10. Zheng X., Vesely, S., Cataland, S., et al. ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8146131/. Accessed Jnauary 6, 2024.
11. Clinical Consult February 12, 2024.

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End Notes

1. 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. [2]
2. 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... [11]

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Revision History

• 2024-02-12: New UM PA criteria.

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