CurrentEmblemHealthPolicy N/A

Adzynma (ADAMTS13, recombinant-krhn) coverage — congenital TTP

Defines accepted indications, contraindications, exclusions, coding, and prior authorization expectations for Adzynma (ADAMTS13, recombinant-krhn) for EmblemHealth members, including adult and pediatric use for congenital TTP and payer documentation/compendia requirements.

Policy Summary

PayerEmblemHealth

PolicyAdzynma (ADAMTS13, recombinant-krhn) coverage — congenital TTP

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization—Evolent will process medication requests and continuation requests may be eligible if criteria for prior use and no disease progression are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

cTTP prophylaxis & on-demandindications described

J7171HCPCS code listed (10 IU)

40 IU/kgsingle dose maximum

Commercial, Exchange, Medicaidapplicable lines of business

Coverage Criteria for Adzynma (ADAMTS13, recombinant-krhn)

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Initial and maintenance therapy for cTTP

Covered when ALL of the following are met

Indication: Confirmed diagnosis of congenital thrombotic thrombocytopenic purpura (cTTP)

Adzynma may be used for prophylactic or on‑demand enzyme replacement therapy in adult and pediatric members

Supporting evidence: Use is supported by FDA labeling, CMS‑approved compendia, NCCN or ASCO clinical guidelines, or acceptable peer‑reviewed literature meeting CMS standards

Evolent requires supporting documentation per policy

Dosing limit: Dosing does not exceed single dose limit of 40 IU/kg<=40 IU/kg per single dose

Requests exceeding this single‑dose limit are excluded

Exclusions: Adzynma (ADAMTS13, recombinant-krhn) is not authorized for treatment of acquired thrombotic thrombocytopenic purpura (acquired TTP), acquired hemolytic uremic syndrome (aHUS), or acquired atypical hemolytic uremic syndrome. Requests in these acquired conditions are specifically excluded from coverage. Additionally, dosing that exceeds a single dose of 40 IU/kg is excluded. Use of Adzynma for investigational or off‑label indications that lack sufficient supporting evidence or are not generally accepted by the medical community is also excluded; insufficient evidence includes lack of representation of the patient or regimen in the published data, absence of clinically meaningful outcomes, or reliance on uncontrolled case reports or abstracts without full peer‑reviewed publication.

Explicit exclusion: Requests for Adzynma to treat acquired TTP, acquired HUS, or acquired atypical HUS are not medically necessary under this guideline and will not be approved. Uses that exceed the single‑dose maximum of 40 IU/kg or that are investigational/off‑label without adequate evidence (as defined by lack of CMS‑recognized compendia support or acceptable peer‑reviewed literature) are also considered not medically necessary.

Prior Authorization and Provider Requirements

Prior Authorization

Prior Authorization Required

Evolent (on behalf of EmblemHealth) requires prior authorization for Adzynma (ADAMTS13, recombinant-krhn). All medication requests must be processed through Evolent; medications not authorized by Evolent may be deemed not approvable and not reimbursable.

Prior authorization required for initial and renewal requests.
Continuation requests may be approved without reapplication to this policy when all continuation conditions are met (see separate continuation criteria).

Note

Provider action — Continuation requests

Providers must ensure continuation requests meet Evolent continuation conditions before submission to avoid unnecessary denials or delays.

Continuation exemption applies only if the member has not experienced disease progression on the medication.
Continuation exemption applies only if the medication was used within the last year without a lapse > 30 days of active authorization.
No additional medications are being added to the continuation request.

Documentation Required

Evidence and documentation requirements

Support for use must be documented according to accepted sources: FDA labeling, CMS‑recognized compendia, NCCN/ASCO clinical guidelines, or peer‑reviewed literature that meets CMS standards. Include relevant citation(s) and clinical rationale in the request.

Attach FDA prescribing information or compendia citation when available (e.g., Adzynma PI).
If relying on peer‑reviewed literature, include full article details and rationale demonstrating applicability to the patient (population, therapy regimen, clinically meaningful outcomes).
Requests for off‑label use must meet CMS Medicare Benefit Policy Manual Chapter 15 criteria and be supported by adequate evidence per policy (see exclusion criteria).

Denial Risk

Denial triggers and exclusion criteria

Requests will be denied for excluded or unsupported uses and for dosing beyond policy limits.

Requests for Adzynma for acquired TTP, acquired HUS, or acquired aHUS are excluded.
Requests with dosing that exceeds a single dose limit of 40 IU/kg will be denied.
Investigational or off‑label indications lacking sufficient evidence (not supported by CMS compendia, guidelines, or acceptable peer‑reviewed literature) are not approvable.

Background

Background: Congenital thrombotic thrombocytopenic purpura (cTTP) is an inherited deficiency of the ADAMTS13 protease that results in accumulation of ultra‑large von Willebrand factor multimers and microvascular platelet‑rich thrombosis. Enzyme replacement with recombinant ADAMTS13 (Adzynma) provides the missing proteolytic activity and may be used as prophylactic or on‑demand enzyme replacement therapy in adults and children with a confirmed diagnosis of cTTP. Clinical use should follow FDA labeling and supporting compendia or peer‑reviewed evidence as specified in the policy.

Key Definitions

cTTP — definition and relation to Adzynma use

DefinitionCongenital Thrombotic Thrombocytopenic Purpura (cTTP) is an inherited ADAMTS13 deficiency for which Adzynma may be used for prophylactic or on‑demand enzyme replacement therapy in adult and pediatric members.

Covered useProphylactic (preventive) or on‑demand enzyme replacement therapy with Adzynma for members with confirmed cTTP diagnosis.

Distinction from acquired diseaseUse of Adzynma for acquired TTP, acquired HUS, or acquired atypical HUS is an exclusion (not covered).