Adzynma (recombinant ADAMTS13) — coverage criteria for congenital TTP
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Defines prior authorization, coverage criteria, dosing, and documentation requirements for Adzynma (recombinant ADAMTS13) used as prophylactic or on‑demand enzyme replacement in congenital thrombotic thrombocytopenic purpura for adult and pediatric patients.
No material clinical or coverage changes in this revision.
Recommended Authorization Criteria
FDA‑Approved Indication — Congenital TTP
Approve for 1 year if the patient meets ALL of the following (A, B, C, and D):
FDA‑Approved Indication criteria
- A) Baseline (prior to therapy) ADAMTS13 activity is < 10% (< 10 IU/dL) [documentation required].< 10% (< 10 IU/dL)
- B) Patient does not have anti‑ADAMTS13 autoantibodies as determined by a diagnostic test [documentation required].
- C) Patient has a pathogenic variant or mutation in the ADAMTS13 gene (typically homozygous or compound heterozygous) [documentation required].
- D) Medication is prescribed by or in consultation with a hematologist.
Dosing
Dosing. Approve the following dosing regimens (A and/or B):
May be approved as ongoing prophylaxis if criteria continue to be met.
Cumulative weekly dosing limit applies.
Coverage of Adzynma for indications or circumstances not specifically listed in the Recommended Authorization Criteria is not recommended. This policy will be updated as new published data become available; providers should not expect approval when the requested use falls outside the criteria set forth in this document.
Use of Adzynma for indications other than those delineated in the Recommended Authorization Criteria is not recommended. Requests for off‑label or non‑listed uses should be considered unsupported by this policy and are unlikely to meet medical necessity for coverage.
Key Laboratory and Code Thresholds
Prior Authorization, Documentation, and Dose Exceptions
Prior Authorization Recommended
Prior authorization is recommended for medical benefit coverage of Adzynma. When criteria are met and the medication is prescribed by or in consultation with a specialist (hematologist), approval is recommended for 1 year. Extended approvals are allowed if the patient continues to meet the Criteria and Dosing.
- Approval duration: up to 1 year when Recommended Authorization Criteria are met
- Prescriber requirement: prescribed by or in consultation with a physician who specializes in the condition (hematologist)
Dose Exception Handling
Requests for doses outside the dosing regimens documented in this policy (routine prophylaxis up to 40 IU/kg IV once weekly; on‑demand therapy up to 135 IU/kg IV per week) will be considered on a case‑by‑case basis by a clinician (e.g., Medical Director or Pharmacist).
- Dose exceptions reviewed by clinician (Medical Director or Pharmacist)
- Document the requested dose, clinical rationale, and supporting records for review
Required Supporting Documentation
Documentation is required to support use of Adzynma as described in the Authorization Criteria. Acceptable documentation includes chart notes, laboratory data (including baseline ADAMTS13 activity), genetic test results (evidence of pathogenic ADAMTS13 variant), and testing demonstrating absence of anti‑ADAMTS13 autoantibodies.
- Baseline ADAMTS13 activity < 10% must be documented prior to therapy
- Documentation of absence of anti‑ADAMTS13 autoantibodies
- Genetic testing showing pathogenic ADAMTS13 variant (homozygous or compound heterozygous)
- Prescriber specialty or consultation note (hematologist)
Non‑Listed Indications Not Recommended for Coverage
Coverage is not recommended for uses not listed in the Recommended Authorization Criteria. Criteria will be updated as new published data become available.
- Do not approve for indications outside the listed criteria
Clinical Background
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare, inherited disorder caused by pathogenic variants in the ADAMTS13 gene resulting in severe hereditary ADAMTS13 deficiency. Clinically, cTTP is characterized by markedly reduced ADAMTS13 activity (< 10% (< 10 IU/dL)), absence of anti‑ADAMTS13 autoantibodies, and a propensity for microvascular thrombosis that can lead to organ damage. The condition most commonly presents in infancy or childhood but may manifest in adulthood or during pregnancy; without appropriate enzyme‑replacement therapy or plasma‑based management, cTTP can be life‑threatening.
Key Definitions
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