CareSource eculizumab prior auth update

Coverage Criteria by Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH) - Initial Therapy

Covered when ALL of the following are met:

PNH initial: Member is at least 18 years of age

PNH initial: Medication is prescribed by or in consultation with a hematologist

PNH initial: Diagnosis of PNH confirmed by flow cytometry

PNH initial: Lactate dehydrogenase (LDH) level >1.5x upper limit of normal (ULN)>1.5x ULN

PNH initial: At least one PNH-related sign/symptom (e.g., fatigue, hemoglobin <10 g/dL, thrombosis, pRBC transfusion, shortness of breath)

PNH initial: Member has tried and failed or is unable to try Ultomiris or Epysqli and Bkemv

Policy prefers Ultomiris

PNH initial: Member has received meningococcal vaccine

PNH initial: Dosage allowed/Quantity limit: 600 mg IV weekly x4, then 900 mg one week later, then 900 mg every 2 weeks

If criteria met, approves for 6 months

Paroxysmal Nocturnal Hemoglobinuria (PNH) - Reauthorization

Covered when ALL of the following are met:

PNH reauth: Clinical evidence of positive response to therapy such as increased hemoglobin level, decreased need for transfusions, normalized LDH levels, or improved fatigue

If met, approve for 12 months

Atypical Hemolytic Uremic Syndrome (aHUS) - Initial Therapy

Covered when ALL of the following are met:

aHUS initial: Medication is prescribed by or in consultation with a hematologist or nephrologist

aHUS initial: Diagnosis of aHUS supported by ALL of the following: (a) thrombocytopenia (platelet count <150 x 10^9/L); (b) evidence of microangiopathic hemolytic anemia (e.g., hemoglobin <10 g/dL, elevated LDH, low haptoglobin, schistocytes); (c) evidence of renal impairment (e.g., raised SCr or low eGFR)

aHUS initial: STEC-HUS has been ruled out

aHUS initial: ADAMTS13 activity level >10% (to rule out TTP)> 10%

Atypical Hemolytic Uremic Syndrome (aHUS) - Reauthorization

Covered when ALL of the following are met:

aHUS reauth: Chart notes demonstrate hematologic normalization (e.g., increased platelet count or LDH maintained below upper limit of normal)

aHUS reauth: Improved or preserved kidney function

If criteria met, approves for 12 months

Generalized Myasthenia Gravis (gMG) - Initial Therapy

Covered when ALL of the following are met:

gMG initial: Member is at least 6 years of age

gMG initial: Medication is prescribed by or in consultation with a neurologist

gMG initial: Documented diagnosis of MGFA class II-IV myasthenia gravis

See Appendix for MGFA classes

gMG initial: Member is seropositive for acetylcholine receptor (AChR) antibodies per lab result

gMG initial:

Generalized Myasthenia Gravis (gMG) - Reauthorization

Covered when ALL of the following are met:

gMG reauth: Chart notes demonstrate improvement in activities of daily living, muscle strength, and/or health-related quality of life; fewer exacerbations or hospitalizations; or reduced use of rescue medication

If criteria met, approves for 12 months

Neuromyelitis Optica Spectrum Disorder (NMOSD) - Initial Therapy

Covered when ALL of the following are met:

NMOSD initial: Member is at least 18 years of age

NMOSD initial: Medication is prescribed by or in consultation with a neurologist

NMOSD initial: Documented diagnosis of NMOSD with seropositivity for aquaporin-4 (AQP4) IgG antibodies

NMOSD initial: One or more relapses within the past year

NMOSD initial: Member has tried and failed rituximab for at least 6 months (requires prior authorization)

Neuromyelitis Optica Spectrum Disorder (NMOSD) - Reauthorization

Covered when ALL of the following are met:

NMOSD reauth: Chart notes document disease stabilization, symptom improvement, and/or reduced frequency of relapses

If criteria met, approves for 12 months

CareSource considers eculizumab (Soliris, Epysqli, Bkemv) not medically necessary for the treatment of conditions that are not listed in this document. For indications not explicitly addressed by this policy, providers should refer to the Off-Label policy for coverage determination.

Any indication not explicitly listed in this policy is considered not medically necessary for eculizumab. Requests for uses outside the listed indications will be evaluated under the Off-Label policy and are not covered unless that review supports medical necessity.

Initial Therapy Requirements

Initial therapy requirements

Initial therapy coverage requires meeting the following indication-specific criteria and program requirements:

Specialist involvement: Medication prescribed by or in consultation with the specialist as specified per indication (hematologist, nephrologist, or neurologist)

See each indication for required specialist

Diagnostic confirmation: Required diagnostic confirmation per indication (e.g., flow cytometry for PNH; ADAMTS13 >10% to exclude TTP for aHUS; AChR seropositivity for gMG; AQP4-IgG seropositivity for NMOSD)

Providers must supply lab results in chart notes

Prior trial of preferred agent: Member has tried and failed or is unable to try Ultomiris (preferred). For NMOSD adults, prior trial and failure of rituximab for at least 6 months is also required

Document trial and reason for inability to try when applicable

Reauthorization / Continuation Criteria

Reauthorization criteria

Reauthorization requires documentation of clinical benefit or disease stability as follows:

PNH continuation: Evidence of clinical response such as increased hemoglobin, decreased transfusion requirement, normalized LDH, or improved fatigue

If met, approve for 12 months

aHUS continuation: Hematologic normalization (e.g., increased platelets or LDH maintained below ULN) and improved or preserved kidney function

If met, approve for 12 months

gMG continuation: Improvement in activities of daily living, muscle strength, health-related quality of life, fewer exacerbations or reduced use of rescue medication

If met, approve for 12 months

Provider Requirements and Operational Rules

Prior Authorization

Prior Authorization Required

Prior authorization is required for eculizumab (Soliris) and its biosimilars Epysqli and Bkemv when billed as a medical benefit. Requests must include supporting clinical documentation and will be reviewed against indication-specific criteria. Approval durations vary by indication (initial approvals typically 6 months; reauthorizations up to 12 months when criteria are met).

Applies to Soliris, Epysqli, Bkemv when billed under the medical benefit
Submit complete chart notes, diagnostic test results, and vaccination records with prior authorization request
Initial approvals generally: 6 months; reauthorization approvals generally: 12 months when renewal criteria met

Step Therapy

Biosimilar / Ultomiris Step Requirement

Step-therapy and biosimilar sequencing requirements apply. In many indications Ultomiris (ravulizumab, preferred) must be tried and failed or the member must have a documented contraindication or intolerance before eculizumab (or its biosimilars) will be approved. For adults with generalized myasthenia gravis, a trial of IV Vyvgart and Ultomiris may be required per indication-specific notes. Document prior trials (drug name, dates, reason for discontinuation or failure) in the chart notes.

Step Therapy Requirements

Step	Requirement	Applicable indications
1	Member must have tried and failed, or be unable to try, Ultomiris (preferred) prior to authorization of eculizumab (Soliris) or its biosimilars (Epysqli, Bkemv)	PNH, aHUS, gMG (adults only for Ultomiris requirement), NMOSD
Exception/documentation	Provider must document reason patient cannot use Ultomiris (e.g., intolerance, contraindication, or lack of access) or provide prior trial and failure documentation	PNH, aHUS, gMG (adults only), NMOSD
Additional NMOSD requirement	For NMOSD, rituximab trial failure for at least 6 months is also required prior to eculizumab authorization	NMOSD
Coverage action	If Ultomiris trial requirement and indication-specific criteria are met, eculizumab or biosimilar may be approved per dosing and duration limits (initial approvals typically 6 months)	PNH, aHUS, gMG, NMOSD
Policy change note	Policy edited to include biosimilars and add biosimilar step edits; Ultomiris designated preferred agent—trial requirement added/clarified	Policy-wide (affects all listed indications)

Coding and Clinical Thresholds

Covered codes (not specified)mixed

N/A	No explicit CPT/HCPCS/NDC codes listed in document; medical benefit implied

LDH threshold (PNH)

LDH threshold>1.5x ULN

ContextRequired for PNH initial authorization as evidence of hemolysis

Approval impactIf present with other criteria, approves eculizumab for 6 months

Platelet count threshold (aHUS)

Platelet count threshold< 150 x 10^9/L

ContextRequired as part of diagnostic support for aHUS initial authorization (thrombocytopenia)

Role

Dosing and Quantity Limits

Eculizumab dosing (PNH)

IndicationPNH dosing (adult) — initiation regimen shown in PNH criteria

Dose and schedule600 mg IV weekly x4, then 900 mg one week later, then 900 mg every 2 weeks

DurationIf criteria met, initial approval for 6 months

Benefit typeMedical benefit (infusion) per policy header

Eculizumab dosing (aHUS - adults)

IndicationaHUS adults

Dose and schedule900 mg IV weekly x4, then 1200 mg one week later, then 1200 mg every 2 weeks

Administration Site

Note

Medical benefit (infusion) administration implied

Eculizumab is administered under the medical benefit (infusion); the policy implies infusion center administration but does not specify a particular site-of-care requirement.

Benefit type is Medical; dosing and quantity limits are provided for IV infusion in the policy.

Biosimilars and Related Policy Notes

Billing Rule

Biosimilars included (Epysqli, Bkemv) and subject to step edits

Epysqli and Bkemv are listed as biosimilars of Soliris and have been added to the policy; the policy was edited to include biosimilars and to implement biosimilar step edits, with Ultomiris noted as preferred in several indications.

Policy title and criteria edited to reference biosimilars and add biosimilar step edits.
Ultomiris preference and trial requirement are stated in the criteria for multiple indications.

Billing Rule

Biosimilars treated like Soliris for step therapy

Biosimilars Epysqli and Bkemv are included in the eculizumab policy and are subject to the same step edits and Ultomiris trial requirements as Soliris.

CareSource edited the policy to add biosimilars and to require trials of preferred agents before authorizing biosimilars.

Background

Eculizumab is a terminal complement (C5) inhibitor used to reduce intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), to inhibit complement‑mediated thrombotic microangiopathy in atypical hemolytic uremic syndrome (aHUS), to treat anti‑AQP4 (AQP4‑IgG) positive neuromyelitis optica spectrum disorder (NMOSD), and to treat anti‑AChR antibody‑positive generalized myasthenia gravis (gMG). Because C5 blockade increases susceptibility to meningococcal infection, meningococcal vaccination is required prior to therapy per the policy.

Definitions

PNH definition and diagnosis

DefinitionParoxysmal nocturnal hemoglobinuria (PNH): a rare hematopoietic stem cell disorder where complement activation destroys red blood cells

Diagnostic methodDiagnosis confirmed by flow cytometry (required for PNH initial authorization)

Associated indicationPNH is an approved indication for eculizumab to reduce hemolysis

aHUS definition

DefinitionAtypical hemolytic uremic syndrome (aHUS): a complement-mediated thrombotic microangiopathy characterized by microangiopathic hemolytic anemia (MAHA), thrombocytopenia, and acute kidney injury/renal impairment

Clinical componentsRequires exclusion of STEC-HUS and differentiation from TTP (ADAMTS13 activity >10%)

Provider-Facing Resources

Documentation Required

Required documentation (provider support / duplicate)

Duplicate: Providers must supply diagnostic confirmation and chart notes demonstrating required laboratory values, antibody seropositivity, specialist consultation, vaccination record, and documentation of prior treatment trials or reasons for inability to try preferred agents; reauthorization requires evidence of clinical benefit or stabilization.

Include vaccination proof (meningococcal) and for aHUS include ADAMTS13 >10% documentation to exclude TTP.
For reauthorization, provide chart notes showing the specific improvement or stabilization required per indication (e.g., normalized LDH, improved platelets, functional gains in gMG, or reduced relapses in NMOSD).

Revision History

2025-06-25title_and_step_editsLatest

Policy title edited to explicitly include biosimilars (Epysqli and Bkemv) and biosimilar step edits were added to require preferred-step trials where specified.

2025-04-07gMG_age_limit_and_label_alignment

Generalized myasthenia gravis (gMG) minimum age changed from at least 18 years to at least 6 years to align with updated FDA labeling; dosing and trial requirements updated and adult-only Ultomiris/Vyvgart trial clarified.

2024-04-10

OpenPayer

Eculizumab (Soliris) and biosimilars (Epysqli, Bkemv) prior authorization and coverage

Coverage Criteria by Indication

Initial Therapy Requirements

Reauthorization / Continuation Criteria

Provider Requirements and Operational Rules

Step Therapy Requirements

Coding and Clinical Thresholds

Dosing and Quantity Limits

Administration Site

Biosimilars and Related Policy Notes

Background

Definitions

Provider-Facing Resources

Revision History