CurrentMass General Brigham Health PlanPolicy N/A

Gamifant (emapalumab-lzsg)

Medical benefit prior authorization policy for Gamifant (emapalumab-lzsg) for treatment of primary hemophagocytic lymphohistiocytosis (HLH) in adult and pediatric patients, including initial and continuation authorization criteria, exclusions, limitations, and documentation requirements.

Policy Summary

PayerMass General Brigham Health Plan

PolicyGamifant (emapalumab-lzsg)

Policy CodePolicy N/A

Change TypeRevisions; formatting and reference updates (no material clinical changes)

Effective DateJul 1, 2025

Next Review Date

Key ActionPrior authorization is required under the medical benefit; submit documentation of specialist prescriber, diagnostic confirmation (molecular or ≥5 clinical criteria), absence of listed infections, prior therapy failure/intolerance, dexamethasone plans, HSCT status, and appropriate dosing.

SourceLink

POLICY UPDATE CHANGES

06/11/25 - Reviewed and updated for P&T. Part of annual UM review. Updated formatting and references. Effective 7/1/25

07/10/24 - Reviewed and updated for P&T. Removed criteria requiring documentation of baseline clinical parameters and lab values as it was repetitive with criterion 3. Effective 8/12/24.

09/21/22 - Created for Sept P&T; matched MH UPPL.

1Covered Indication (primary HLH)

6 monthsApproval Duration

8HLH Diagnostic Criteria Items Listed

Coverage Summary

Coverage stance: covered_with_criteria for Gamifant (emapalumab-lzsg). Scope summary: Medical benefit prior authorization policy for Gamifant (emapalumab-lzsg) for treatment of primary hemophagocytic lymphohistiocytosis (HLH) in adult and pediatric patients, including initial and continuation authorization criteria, exclusions, limitations, and documentation requirements. Subject: Gamifant (emapalumab-lzsg). Payer and benefit: MassHealth — medical benefit prior authorization. Effective date: 07/01/2025. Last review date: 06/11/2025.

Policy highlights

Approval Duration6 months (initial and reauthorizations)

Covered IndicationPrimary hemophagocytic lymphohistiocytosis (HLH)

HLH diagnostic items count8 diagnostic items listed for primary HLH (fever, splenomegaly, cytopenias, hypertriglyceridemia/hypofibrinogenemia, hemophagocytosis, low/absent NK activity, ferritin ≥500, soluble CD25 ≥2400)

StatusCURRENT

Confidence / NotesPolicy effective 07/01/2025; prior reviews documented (created 09/21/22; updates 07/10/24, 06/11/25)

Initial Authorization Criteria

Initial Authorization (OR authorization path)

Authorization may be granted when ALL the following criteria are met, and documentation is provided:

ALL of the following

Diagnosis and prescriber
- Diagnosis primary hemophagocytic lymphohistiocytosis (HLH)
- Prescriber is a specialist (e.g., hematologist and oncologist) or consult notes from a specialist are provided
ONE of the following (diagnostic confirmation)
- Molecular confirmation: Molecular tests confirming diagnosis of primary HLH
- At least five of the following suggesting primary HLH
  - Fever
  - Splenomegaly
  - Cytopenias defined by 2 of the following
    Hemoglobin: hemoglobin < 9 g/dLhemoglobin < 9 g/dL
    Platelets: platelets < 100 x 10^9 /Lplatelets < 100 x 10^9 /L
Active disease: Member has active disease
Infection exclusions: Member does not have active infections caused by specific pathogens favored by IFNγ neutralization (e.g., mycobacteria, Histoplasma Capsulatum, Shigella, salmonella, campylobacter, leishmanial infections)
Prior conventional therapy: Inadequate response, adverse reaction or contraindication to conventional HLH therapy (chemotherapy and/or systemic corticosteroids and/or immunosuppressive therapy)
Conventional HLH therapy examples listed in policy
ONE of the following (dexamethasone)
- Dexamethasone concurrent: Dexamethasone will be administered concurrently
- Dexamethasone rationale: Clinical rationale for not using dexamethasone
ONE of the following (HSCT status)
- Anticipated HSCT (haematopoietic stem cell transplantation) date
- Not candidate HSCT: Member is not a candidate for HSCT
Dosing: Appropriate dosing

Continuation of Therapy Criteria (Reauthorization)

Continuation of Therapy (Reauthorization)

Prescriber provides documentation of ALL of the following:

ALL of the following

Positive response to therapy evidenced by ONE of the following
- Complete response: normalization of all HLH abnormalities
- Partial response: normalization of ≥ 3 HLH abnormalities≥ 3 abnormalities normalized
- Improvement: HLH improvement: ≥ 3 HLH abnormalities improved by at least 50% from baseline≥ 3 abnormalities improved ≥ 50% from baseline
ONE of the following (dexamethasone)
- Dexamethasone concurrent: Dexamethasone will be administered concurrently
- Dexamethasone rationale: Clinical rationale for not using dexamethasone
ONE of the following (HSCT status)
- Anticipated HSCT date
- Not candidate HSCT: Member is not a candidate for HSCT

Recertification thresholds (continuation)

Ferritin (recertification)Ferritin < 2,000 mcg/L

Fibrinogen (recertification)Fibrinogen > 1.5 g/L

D-dimer (recertification)D-dimer < 500 mcg/mL

Soluble CD25 (recertification)Soluble CD25 > 2-fold from baseline

ContextThese are listed among HLH abnormalities/normalization values evaluated for continuation (positive response definitions in continuation criteria)

Exclusions and Infection-related Contraindications

Exclusion: Authorization requires that the member does not have active infections caused by specific pathogens favored by IFNγ neutralization; examples listed include mycobacteria, Histoplasma capsulatum, Shigella, salmonella, campylobacter, and leishmanial infections. The policy lists documentation of absence of these infections as a required documentation item for authorization.

Provider Actions and Documentation Requirements

Prior Authorization

Prior authorization required

Prior authorization is required under the medical benefit (MassHealth). Authorization may be reviewed case-by-case for members new to the plan who are currently receiving treatment.

Documentation Required

Specialist prescriber or consult notes required

Prescriber must be a specialist (e.g., hematologist/oncologist) or consult notes from a specialist must be provided.

Documentation Required

Diagnostic confirmation or 5-of-8 clinical criteria required

Provide either molecular confirmation of primary HLH OR documentation showing at least five of the following diagnostic features. Cytopenias require at least two of: hemoglobin < 9 g/dL; platelets < 100 x 10^9/L; neutrophils < 1 x 10^9/L. Other criteria include fever, splenomegaly, hypertriglyceridemia (fasting triglyceride ≥ 265 mg/dL or > 3 mmol/L) and/or fibrinogen ≤ 150 mg/dL, hemophagocytosis in marrow/spleen/lymph nodes, low/absent NK cell activity, ferritin ≥ 500 mcg/L, or soluble CD25 ≥ 2400 U/mL.

Documentation Required

Infection exclusion documentation

Document that the member does not have active infections caused by pathogens favored by IFNγ neutralization (examples: mycobacteria, Histoplasma capsulatum, Shigella, salmonella, campylobacter, leishmanial infections).

Documentation Required

Document prior failure/intolerance to conventional HLH therapy

Provide documentation of inadequate response, adverse reaction, or contraindication to conventional HLH therapy (chemotherapy and/or systemic corticosteroids and/or immunosuppressive therapy); examples of conventional regimens are listed in the policy appendix.

Documentation Required

Dexamethasone concurrent use or rationale

Document that dexamethasone will be administered concurrently, or provide clinical rationale for not using dexamethasone.

Documentation Required

HSCT planning documentation

Provide anticipated hematopoietic stem cell transplantation (HSCT) date or documentation that the member is not a candidate for HSCT.

Documentation Required

Continuation response documentation

For reauthorization, document a positive response as defined by the policy (complete response: normalization of all HLH abnormalities; partial response: normalization of ≥ 3 HLH abnormalities; or HLH improvement: ≥ 3 HLH abnormalities improved by ≥ 50% from baseline) and provide associated laboratory and clinical parameter changes.

Billing Rule

Approval duration

Initial approvals and reauthorizations are limited to 6 months.

Clinical Evidence and References

Evidence cited in the policy includes: Gamifant [package insert] Waltham (MA): Sobi Inc; 2024 Oct. and the key clinical trial Locatelli F et al., Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis, N Engl J Med. 2020 May 7;. These references are listed in the policy; no additional trial metrics are provided in the policy text.

Background and Definitions

Background/indication: Gamifant (emapalumab-lzsg) is an interferon gamma (IFNγ) blocking antibody indicated for treatment of adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance to conventional HLH therapy. Policy governance: this document governs medical-benefit prior authorization for MassHealth members requesting Gamifant; prior authorization is required and may be reviewed case-by-case for members new to the plan currently receiving treatment.

Term	Definition
Active disease
Not explicitly defined in policy; implies ongoing clinical or laboratory evidence of HLH requiring therapy.
Conventional HLH therapy
Chemotherapy and/or systemic corticosteroids and/or immunosuppressive therapy; examples listed (e.g., etoposide+dexamethasone, CHOP, DEP).

Revision History

09/21/22created

Created for Sept P&T; matched MassHealth UPPL.

07/10/24updated

Removed redundant baseline documentation requirement (not material to clinical policy statements); reviewed and updated for P&T. Effective 8/12/24.

06/11/25updatedLatest

Formatting and references updated (not material to clinical policy statements); reviewed and updated for P&T as part of annual UM review. Effective 7/01/25.

Policy Summary

PayerMass General Brigham Health Plan

PolicyGamifant (emapalumab-lzsg)

Policy CodePolicy N/A

Change TypeRevisions; formatting and reference updates (no material clinical changes)

Effective DateJul 1, 2025

Next Review Date

SourceLink

On This Page

OpenPayer

Gamifant (emapalumab-lzsg)

Coverage Summary

Initial Authorization Criteria

Continuation of Therapy Criteria (Reauthorization)

Limitations and Approval Duration

Exclusions and Infection-related Contraindications

Applicable Codes

Provider Actions and Documentation Requirements

Clinical Evidence and References

Background and Definitions

Revision History