CurrentUnitedHealthcarePolicy 2025D0131D

Adzynma (ADAMTS13, recombinant-krhn) for congenital thrombotic thrombocytopenic purpura

Defines UnitedHealthcare coverage criteria for prophylactic and on-demand use of Adzynma in patients with congenital thrombotic thrombocytopenic purpura (cTTP), including authorization durations and prescribing requirements.

Policy Summary

PayerUnitedHealthcare

PolicyAdzynma (ADAMTS13, recombinant-krhn) for congenital TTP

Policy CodePolicy 2025D0131D

Change TypeRevised (updated Benefit Considerations and References)

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and document confirmed ADAMTS13 mutation by molecular genetic testing, hematologist involvement, indication, and FDA-consistent dosing.

SourceLink

POLICY UPDATE CHANGES

Updated Benefit Considerations and References sections to reflect the most current information.

12 momax prophylaxis auth

3 momax on-demand auth

46study patients

0acute events on therapy

Coverage Criteria for Adzynma (ADAMTS13, recombinant-krhn)

Initial Prophylactic Therapy

Covered when ALL of the following are met for initial prophylactic therapy:

Initial prophylactic therapy criteria: Diagnosis of congenital thrombotic thrombocytopenic purpura (cTTP); ADAMTS13 mutation confirmed by molecular genetic testing; prescribed for routine prophylactic treatment to prevent thrombotic thrombocytopenic purpura (TTP) events; Adzynma dosing is in accordance with the United States Food and Drug Administration approved labeling; prescribed by or in consultation with a hematologist; authorization will be for no more than 12 months

Continuation Therapy

Covered when ALL of the following are met for continuation of prophylactic therapy:

Continuation therapy criteria: Patient has previously received routine prophylactic treatment with Adzynma; documentation of positive clinical response to Adzynma therapy; Adzynma dosing is in accordance with the United States Food and Drug Administration approved labeling; prescribed by or in consultation with a hematologist; authorization will be for no more than 12 months

Policy Summary

PayerUnitedHealthcare

PolicyAdzynma (ADAMTS13, recombinant-krhn) for congenital TTP

Policy CodePolicy 2025D0131D

Change TypeRevised (updated Benefit Considerations and References)

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and document confirmed ADAMTS13 mutation by molecular genetic testing, hematologist involvement, indication, and FDA-consistent dosing.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

When the requirements listed in the coverage criteria are not fully met, the request for Adzynma (ADAMTS13, recombinant-krhn) will not satisfy UnitedHealthcare’s coverage conditions. There are no separate, explicit non-coverage exclusions beyond failure to meet the specified criteria. Coverage is contingent on meeting all applicable elements for the intended use (diagnosis of congenital TTP, molecular genetic confirmation of an ADAMTS13 mutation, indication-specific dosing per FDA labeling, hematologist involvement, and the applicable authorization duration).

In other words, absence of any required criterion — for example, missing genetic confirmation, dosing that does not follow the United States Food and Drug Administration approved labeling, or lack of prescribing/consultation with a hematologist — means the request does not meet the policy’s conditions for medical necessity.

Examples of uses that would not meet the coverage criteria include, but are not limited to: no confirmed ADAMTS13 mutation by molecular genetic testing; dosing that is not in accordance with the United States Food and Drug Administration approved labeling; and therapy that is not prescribed by or in consultation with a hematologist.

Other situations that fail to meet the policy requirements include requests lacking documentation of the intended indication (routine prophylactic versus on‑demand for an acute TTP event), absence of prior receipt/positive response documentation when requesting continuation of prophylaxis, or authorization requests exceeding the policy’s maximum durations (12 months for prophylactic therapy, 3 months for on‑demand treatment).

Provider Requirements, Documentation, and Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required for Adzynma (ADAMTS13, recombinant-krhn). Authorizations for routine prophylactic use will be issued for up to 12 months. Authorizations for on‑demand treatment of an acute TTP event will be issued for up to 3 months. Adzynma must be prescribed by or in consultation with a hematologist.

Authorization durations: prophylactic — up to 12 months; on‑demand — up to 3 months.
Prescriber requirement: prescribed by or in consultation with a hematologist.
Dosing must follow FDA‑approved labeling.

Documentation Required

Documentation Requirements

Documentation must demonstrate the diagnosis, molecular confirmation of the ADAMTS13 mutation, the treatment indication (routine prophylactic vs on‑demand for an acute TTP event), dosing consistent with FDA labeling, and prescriber/consultation details. For continuation requests, documentation must also show prior routine prophylactic use and a positive clinical response to Adzynma.

Required documentation: diagnosis of congenital TTP (cTTP).
Molecular genetic testing confirming ADAMTS13 mutation.
Indication: routine prophylactic or on‑demand acute TTP event.
Dosing documentation consistent with FDA labeling.
Prescriber: hematologist or documented hematology consultation.
For continuation: prior prophylactic Adzynma use and evidence of positive clinical response.

Denial Risk

Triggers for Denial

Requests will be denied if required clinical or administrative elements are missing. Common triggers for denial include absence of confirmed ADAMTS13 mutation, lack of hematologist prescription or documented consultation, indications that do not match prophylactic vs on‑demand criteria, or dosing that is inconsistent with FDA‑approved labeling.

No molecular genetic confirmation of ADAMTS13 mutation.
Prescriber is not a hematologist and no documented hematology consultation.
Indication not clearly for routine prophylactic use or on‑demand treatment of an acute TTP event.
Dosing not consistent with FDA‑approved labeling.
For continuation requests: lack of documented prior prophylactic use or absence of documented positive clinical response.

Step Therapy

Step‑Therapy / Clinical Trial Context

Policy position and clinical trial context: ADAMTS13, recombinant‑krhn was compared to plasma‑based therapies in a randomized, active‑controlled crossover trial (Study 1). The trial demonstrated efficacy of prophylactic ADAMTS13 in preventing acute TTP events versus plasma; this clinical evidence supports the policy’s coverage criteria and prior authorization requirements.

Study 1: global, prospective, randomized, active‑controlled, open‑label, multicenter, two‑period crossover study with a single‑arm continuation period.
Efficacy finding: no acute TTP events occurred in patients receiving ADAMTS13 prophylaxis during the study periods.

Key Definitions

Prophylactic treatment — definition

DefinitionRoutine administration of Adzynma (ADAMTS13, recombinant-krhn) to prevent thrombotic thrombocytopenic purpura (TTP) events in patients with congenital TTP (cTTP).

Required diagnostic confirmationDiagnosis of congenital thrombotic thrombocytopenic purpura (cTTP) with ADAMTS13 mutation confirmed by molecular genetic testing.

Prescriber requirementPrescribed by or in consultation with a hematologist.

Dosing requirementDosing must be in accordance with United States Food and Drug Administration approved labeling.

Authorization durationAuthorization will be for no more than 12 months.

On-demand treatment — definition

DefinitionAdministration of Adzynma (ADAMTS13, recombinant-krhn) for treatment of an acute thrombotic thrombocytopenic purpura (TTP) event in patients with congenital TTP (cTTP).

Required diagnostic confirmationDiagnosis of congenital thrombotic thrombocytopenic purpura (cTTP) with ADAMTS13 mutation confirmed by molecular genetic testing.

Prescriber requirementPrescribed by or in consultation with a hematologist.

Dosing requirementDosing must be in accordance with United States Food and Drug Administration approved labeling.

Authorization durationAuthorization will be for no more than 3 months.

Background and Clinical Context

Congenital thrombotic thrombocytopenic purpura (cTTP) is a hereditary disorder caused by severe deficiency of the ADAMTS13 enzyme, which predisposes patients to recurrent thrombotic thrombocytopenic purpura (TTP) events. Recombinant ADAMTS13 (Adzynma, ADAMTS13, recombinant-krhn) is an enzyme replacement therapy developed to restore ADAMTS13 activity and prevent or treat acute TTP episodes in patients with genetically confirmed cTTP.

Clinical development included a global, prospective, randomized, active‑controlled study comparing recombinant ADAMTS13 to plasma‑based therapies; prophylactic administration of ADAMTS13 demonstrated prevention of acute TTP events in the pivotal study. Within this policy, Adzynma is positioned for both routine prophylactic use and for on‑demand treatment of acute TTP events in patients who meet the specified diagnostic, genetic, dosing, and prescriber criteria.

OpenPayer

Adzynma (ADAMTS13, recombinant-krhn) for congenital thrombotic thrombocytopenic purpura

Coverage Criteria for Adzynma (ADAMTS13, recombinant-krhn)

Provider Requirements, Documentation, and Authorization

Key Definitions

Background and Clinical Context

Policy Revision History