CurrentCentenePolicy N/A

ADAMTS13, Recombinant-krhn (Adzynma)

Defines medical necessity criteria, dosing limits, durations, approvals and exclusions for Adzynma (rADAMTS13) for prophylactic and on-demand treatment of congenital thrombotic thrombocytopenic purpura (cTTP) across Commercial, HIM, and Medicaid lines of business.

Policy Summary

PayerCentene

PolicyADAMTS13, Recombinant-krhn (Adzynma)

Policy CodePolicy N/A

Change TypeFDA label updatecoding revisionsapproval duration clarified

Effective Date11.09.23

Next Review Date

Key ActionProvider must submit documentation (office notes, lab results, genetic test results) supporting that member meets all approval criteria, including genetic confirmation and ADAMTS13 activity, and include current body weight in kg for dosing; justify acute dosing beyond 2 weeks.

SourceLink

POLICY UPDATE CHANGES

Policy updated per FDA labeling to add age ≥ 2 years and define acute TTP event criteria, require documentation of body weight in kg, update maximum dosing, and set prophylaxis initial approval to 6 months and continued to 12 months.

HCPCS/CPT code list revised: added C9167, later added J7171 and removed J3590 and C93991/C91671 in sequence.

Approval durations clarified: prophylaxis initial 6 months and continued 12 months; acute treatment up to 2 weeks per episode.

Policy created pre-emptively on 2023-07-11 with P&T approval 08.23 and subsequent updates recorded 12.04.23 and 02.25 (P&T Approval).

cTTPCovered Indications

Acquired/immune TTP

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40 IU/kgProphylaxis Max Dose

40 IU/kgAcute Max Dose (day1)

Coverage Summary

Adzynma (rADAMTS13) is a human recombinant ADAMTS13 indicated for prophylactic or on-demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). Therapeutic alternatives include plasma infusion products (e.g., fresh frozen plasma, solvent/detergent plasma) and therapeutic plasma exchange. Coverage stance: covered_with_criteria, with medical necessity criteria, dosing limits, approval durations, and exclusions defined in the policy.

Key thresholds and quick facts

Covered IndicationscTTP (congenital)

Not CoveredAcquired/immune TTP

Minimum Age≥ 2 years

Prophylaxis Max Dose40 IU/kg once weekly (must not exceed)

Acute Max Dose (day 1)40 IU/kg on day 1 (then 20 IU/kg day 2; 15 IU/kg/day thereafter)

ADAMTS13 activity threshold≤ 10% of normal

Initial Therapy Criteria

Initial Approval Criteria

Adzynma is medically necessary when ALL of the following are met:

ALL of the following

cTTP diagnostic requirements
- Genetic test confirming biallelic ADAMTS13 mutation
- ADAMTS13 activity ≤ 10% of normal, unless member is currently receiving prophylactic plasma therapy≤ 10% of normal
- Absence of inhibitor/antibody
  - Absence of ADAMTS13 functional inhibitor

Continuation Therapy Criteria

Continued Therapy Criteria

Member must meet ONE of the following:

ALL of the following

ANY of the following
- Member is currently receiving medication via Centene benefit OR member has previously met initial approval criteria
Member is responding positively to therapy as evidenced by improvement in any of the following: thrombocytopenia, microangiopathic hemolytic anemia, or symptom improvement (e.g., fewer headaches, less lethargy, and/or less abdominal pain)
Documentation of member's current body weight (in kg)
Dose and duration limits for continued therapy

Unapproved Indications / Exclusions

Diagnoses/Indications Not Authorized

ANY of the following

Acquired/immune thrombotic thrombocytopenic purpura
Non–FDA approved indications not addressed in this policy unless sufficient documentation per off-label use policies (CP.CPA.09 commercial, HIM.PA.154 marketplace, CP.PMN.53 Medicaid)

Denial Risk

Exclusion for acquired TTP

Coverage is not authorized for acquired/immune thrombotic thrombocytopenic purpura (acquired TTP). Claims or requests for Adzynma for acquired/immune TTP may be denied per policy.

Provider Actions

Prior Authorization

Prior authorization required

Prior authorization is required. Requests will be reviewed against the policy criteria and require documentation of the member's current body weight in kilograms for dose calculation. Providers must justify continued acute dosing if treatment exceeds 2 weeks.

Affected codes: J7171
Affected codes: C9167
Provider justification required for acute dosing > 2 weeks

Documentation Required

Support clinical documentation

Submit supporting clinical documentation with the request, including office notes and laboratory results. Documentation must confirm genetic test results showing biallelic ADAMTS13 mutation and ADAMTS13 activity to demonstrate eligibility.

Genetic test confirming biallelic ADAMTS13 mutation

Applicable Codes

HCPCS Codes referencedHCPCS

C9167	ADAMTS13, recombinant-krhn (historically added)
J7171	ADAMTS13, recombinant-krhn (current HCPCS added 06.03.24)
J3590	Unlisted drug - removed
C9399	Unclassified - removed (C93991 noted as removed in document)

Clinical Evidence

2–4 daysAdzynma pivotal trial: acute episode duration range

~7 daysInternational Hereditary TTP Registry: median acute episode duration

Key evidence includes the Adzynma pivotal trial, which reported acute episode durations ranging 2–4 days, and data from the International Hereditary TTP Registry reporting a median acute episode duration of approximately 7 days. The policy incorporates FDA labeling updates (including age expansion to ≥ 2 years and defined criteria for an acute TTP event) and reflected those changes in approval durations and dosing requirements.

Background

Adzynma is a human recombinant A disintegrin and metalloproteinase with thrombospondin motifs 13 (rADAMTS13) indicated for prophylactic or on‑demand enzyme replacement therapy in congenital TTP (cTTP). Therapeutic alternatives include plasma infusion (fresh frozen plasma, solvent/detergent plasma, thawed plasma) and therapeutic plasma exchange.

Glossary: cTTP = congenital thrombotic thrombocytopenic purpura; ADAMTS13 = A disintegrin and metalloproteinase with thrombospondin motifs 13; LDH = lactate dehydrogenase.

Revision History

2023-07-11Administrative

Policy created pre-emptively (P&T approval 08.23).

2023-11-09Administrative

Effective date established for clinical policy for ADAMTS13, recombinant-krhn (Adzynma).

2023-12-04Material

Policy updated per FDA labeling: added age ≥ 2 years, defined acute TTP event criteria for on-demand therapy, required documentation of body weight (kg) for dose calculation, updated FDA-labeled maximum dosing, set prophylaxis initial approval to 6 months and continued to 12 months, and added provider justification requirement for continued acute dosing exceeding 2 weeks.

Policy Summary

PayerCentene

PolicyADAMTS13, Recombinant-krhn (Adzynma)

Policy CodePolicy N/A

Change TypeFDA label updatecoding revisionsapproval duration clarified

Effective Date11.09.23

Next Review Date

SourceLink

On This Page

Absence of anti-ADAMTS13 antibodies

Age ≥ 2 years≥ 2 years

Prescribed by or in consultation with a hematologist

Documentation of member's current body weight (in kg) for dose calculation

Failure of plasma therapy or contraindication

Failure of plasma therapy (e.g., plasma infusion, therapeutic plasma exchange), unless contraindicated or clinically significant adverse effects are experienced

Indication type

ALL of the following
- Platelet count < 100,000/µL or a drop in platelet count ≥ 50% of the baseline platelet count< 100,000/µL OR ≥ 50% drop from baseline
- Microangiopathic hemolytic anemia with lactate dehydrogenase (LDH) elevation > 2x baseline or > 2x upper limit of normalLDH > 2x baseline or > 2x ULN
For prophylaxis requests: member has TTP signs or symptoms that are persistent or recurrent (examples include persistent thrombocytopenia, recurrent microangiopathic hemolytic anemia, proteinuria, stroke, TIA, lethargy, headaches, loss of concentration, abdominal discomfort)

Dose limits (must not exceed)

For prophylactic therapy: 40 IU/kg once weekly≤ 40 IU/kg once weekly
For acute treatment: 40 IU/kg on day 1, followed by 20 IU/kg on day 2, and 15 IU/kg/day thereafter until two days after the acute event is resolvedDay1 40 IU/kg; Day2 20 IU/kg; Day3+ 15 IU/kg/day until 2 days after resolution

Approval Duration - Initial

ANY of the following

Prophylaxis: 6 months
Acute treatment: Up to 2 weeks per acute episode

For prophylactic therapy: 40 IU/kg once weekly (dose increase requests must not exceed this)≤ 40 IU/kg once weekly

Acute continued therapy

For acute treatment: dosing per initial acute regimen (40 IU/kg day 1; 20 IU/kg day 2; 15 IU/kg/day until two days after resolution)
If request exceeds 2 weeks of treatment: provider justification for continued acute dosing is required

ADAMTS13 activity level (e.g., ≤ 10% of normal)

Relevant labs and office notes (platelets, LDH, clinical assessment)

Billing Rule

Dose limit enforcement

Dose limits are enforced per labeling. Requests must not exceed the labeled maximums for prophylaxis or the acute/on‑demand regimen.

Prophylaxis: ≤ 40 IU/kg once weekly
Acute Day 1: 40 IU/kg
Acute Day 2: 20 IU/kg
Acute Day 3 and thereafter: 15 IU/kg/day (until two days after resolution)

2024-06-03Material

HCPCS code changes: added J7171 on 06.03.24 and removed prior HCPCS entries (sequence of additions/removals including earlier addition of C9167 and removals of J3590 and C93991/C91671).

2025-02-25AdministrativeLatest

1Q 2025 annual review (P&T Approval 02.25): no significant clinical policy statement changes; references reviewed and updated.