CurrentMass General Brigham Health PlanPolicy N/A

Prior authorization for specialty pharmacy medication for acute hereditary angioedema (HAE) attacks

Prior authorization policy for coverage of a specialty pharmacy medication used to treat acute hereditary angioedema (HAE) attacks in commercial/exchange members; governs pharmacy benefit processing and specialty fill requirements.

Policy Summary

PayerMass General Brigham Health Plan

PolicyPrior authorization for specialty pharmacy medication for acute hereditary angioedema (HAE) attacks

Policy CodePolicy N/A

Change TypeBenefit designation revised to Pharmacy Benefit

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionObtain prior authorization for pharmacy specialty fills; approvals are for 6 months and continuation requires documented clinical benefit.

SourceLink

POLICY UPDATE CHANGES

Policy updated to indicate it no longer applies to the medical benefit and is under the pharmacy benefit only.

6 monthsapproval duration for authorizations

Commercial/Exchangeplan applicability

Prior Authorizationprogram type

Specialty pharmacyfill site requirement

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Initial Authorization Criteria

Authorization may be granted when the medication will be used alone for acute HAE attacks and ONE of the following two main pathways is met:

Overall: Medication will not be used in combination with any other medication used for acute HAE attacks AND (Pathway A OR Pathway B)

Pathway A: C1-INH deficiency/dysfunction: Member has C1 inhibitor deficiency or dysfunction as confirmed by laboratory testing

AND both: (1) C4 below lab lower limit of normal; and (2) either C1-INH antigenic level below lab lower limit OR C1-INH functional level <50% or below lab lower limit

Pathway B: Normal C1-INH with other evidence: Member has normal C1 inhibitor as confirmed by laboratory testing

AND either: (1) pathogenic mutation in F12, angiopoietin-1, plasminogen, KNG1, HS3ST6, or MYOF confirmed by genetic testing; OR (2) documented family history of angioedema AND angioedema refractory to trial of high-dose antihistamine (cetirizine 40 mg/day or equivalent) for >=1 month

Continuation Therapy

Continuation may be granted when ALL of the following are met:

Continuation: (A) Member meets criteria for initial approval; (B) Member has experienced a reduction in severity and/or duration of attacks when the medication was used for an acute attack; (C) Prophylaxis should be considered based on attack frequency, severity, comorbid conditions, and member's quality of life.

All indications other than the treatment of acute hereditary angioedema (HAE) attacks are considered experimental/investigational and not medically necessary.

Use of this medication for indications other than acute HAE attacks — including uses not supported by the FDA‑approved indication or other off‑label uses — is not medically necessary and will not be covered. The FDA label also notes that effectiveness was not established in HAE patients with laryngeal attacks.

Provider Requirements, Prior Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for pharmacy benefit specialty fills. All approvals are granted for 6 months; continuation requires meeting the continuation criteria listed below.

Specialty medication — must be filled at a contracted specialty pharmacy.
Contact (Pharmacy Benefit): Phone: 800-711-4555; Fax: 844-403-1029
Contact (Medical Benefit): Phone: 833-895-2611; Fax: 888-656-6671
Approval duration: 6 months

Denial Risk

Indication Restrictions

Use for indications other than treatment of acute hereditary angioedema (HAE) attacks (and laryngeal attacks where effectiveness was not established) is considered experimental/investigational or not medically necessary.

FDA-approved indication: Treatment of acute attacks in adults and adolescent patients with HAE. Limitation: effectiveness not established in laryngeal attacks.
All other indications are experimental/investigational or not medically necessary.

Documentation Required

Required Supporting Tests and History

Laboratory confirmation and/or genetic testing documentation is required to support the diagnosis and indicate appropriate use.

Required labs for C1-INH deficiency/dysfunction: C4 below lab lower limit of normal AND either C1-INH antigenic level below lab lower limit of normal OR C1-INH functional level <50% (or below lab lower limit of normal).
If C1-INH is normal, require genetic testing showing mutation in F12, ANGPT1 (angiopoietin-1), PLG (plasminogen), KNG1, HS3ST6, MYOF, or other relevant gene, OR documented family history of angioedema with refractoriness to high‑dose antihistamine therapy (e.g., cetirizine 40 mg/day or equivalent for ≥1 month).
New-to-plan continuity: members new to the plan within the past 90 days who are currently receiving the medication (excluding samples or manufacturer patient assistance programs) may be authorized.

Documentation Required

Response Documentation for Continuation

For continuation of therapy, documentation must show clinical benefit when the medication was used for acute attacks.

Continuation criteria: member meets initial approval criteria and has experienced a reduction in severity and/or duration of attacks when treated with the requested medication.
Consideration: prophylaxis decisions should be based on attack frequency, severity, comorbid conditions, and member quality of life.

Step Therapy

Step Therapy and Combination Therapy

No step therapy requirements apply for acute treatment in this policy; however, combination acute therapy with other acute HAE medications is not permitted.

Step therapy: Not applicable for acute HAE treatment under this policy.
Do not authorize requests where the medication will be used in combination with any other medication for acute HAE attacks — such requests may be denied.

Definitions and Diagnostic Criteria

C1‑INH deficiency/dysfunction (laboratory definition)

Diagnostic criteriaC1-INH deficiency/dysfunction is defined by: C4 below the laboratory lower limit of normal AND either (a) C1‑INH antigenic level below the laboratory lower limit OR (b) normal C1‑INH antigenic level with C1‑INH functional level <50% or below the laboratory lower limit of normal.

Required laboratory test 1Serum C4 level: result below the laboratory lower limit of normal.

Required laboratory test 2C1‑INH antigenic (quantitative) level: below the laboratory lower limit of normal fulfills the requirement.

Alternate functional criterionIf C1‑INH antigenic level is normal, a low C1‑INH functional assay (functional level <50% or below the laboratory lower limit) qualifies.

HAE with normal C1‑INH (diagnostic support)

Genetic confirmationPathogenic mutation in one of the following genes confirmed by genetic testing: F12, angiopoietin‑1, plasminogen, KNG1, HS3ST6, or MYOF.

Clinical alternativeDocumented family history of angioedema AND angioedema refractory to high‑dose antihistamine (e.g., cetirizine 40 mg/day or equivalent) for at least one month.

Laboratory status requirementNormal C1‑INH as confirmed by laboratory testing is required for this pathway (i.e., HAE with normal C1‑INH).

OpenPayer

Prior authorization for specialty pharmacy medication for acute hereditary angioedema (HAE) attacks

Coverage and Medical Necessity Criteria

Provider Requirements, Prior Authorization, and Documentation

Clinical Background

Definitions and Diagnostic Criteria