CurrentNeighborhood Health Plan of Rhode IslandPolicy Evolent Clinical Guideline 3127 for Fabhalta TM (iptacopan)

Evolent Clinical Guideline 3127 for Fabhalta TM (iptacopan)

Defines Evolent's coverage and prior authorization requirements for Fabhalta (iptacopan) in adults with PNH, including acceptable evidence sources and continuity rules; applies to providers submitting medication requests to Evolent.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyFabhalta (iptacopan) coverage criteria for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Policy CodePolicy Evolent Clinical Guideline 3127 for Fabhalta TM (iptacopan)

Change TypeTemplate conversion and revisions to indications/exclusions

Effective DateJun 1, 2024

Next Review Date

Key ActionSubmit prior authorization requests for Fabhalta (iptacopan) and document FDA labeling or CMS-recognized compendia support, ensure REMS enrollment and vaccination for encapsulated bacteria prior to initiation.

SourceLink

POLICY UPDATE CHANGES

Converted to new Evolent guideline template and replaced UM ONC_1501 Fabhalta (iptacopan).

Updated indication section and updated exclusion criteria including maximum dosage form quantities.

1Covered indication (PNH)

J8999Billing code listed

200 mgSingle dose maximum

60 caps

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Coverage Criteria for Fabhalta (iptacopan)

Initial Therapy

Covered when ALL of the following are met

Covered indication: Patient is an adult with paroxysmal nocturnal hemoglobinuria (PNH)

From indication section

Evidence support: Use is supported by FDA approved product labeling, CMS-approved compendia, NCCN or ASCO clinical guidelines, or acceptable peer-reviewed literature

Accepted evidence sources required per policy

REMS and vaccination: Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose unless the risks of delaying therapy outweigh the risk of infection; comply with the FABHALTA REMS

Boxed warning and REMS requirement

Dose limits: Single dose does not exceed 200 mg and monthly total does not exceed 60 (200 mg) capsulesSingle dose <= 200 mg; Monthly <= 60 capsules (200 mg)

From dosing and exclusion limits

Continuation Therapy

Covered when ALL of the following are met

No disease progression: Member has not experienced disease progression while taking Fabhalta

Continuation exemption applies

Recent prior authorization: Requested medication was used within the last year without a lapse of more than 30 days of having an active authorization<= 30 day lapse; prior use within 1 year

Continuation rule

No new medications: No additional medications are being added to the continuation request

Continuation rule

The following situations are explicit exclusions to coverage for Fabhalta (iptacopan): disease progression while taking Fabhalta, and concurrent use with other therapies used to treat PNH (for example, eculizumab or ravulizumab). Dosing that exceeds the policy’s limits is also excluded — specifically, any single dose that exceeds 200 mg or cumulative treatment that exceeds 60 capsules per month (200 mg capsules) will trigger denial. These exclusions reflect that progression on therapy or combination use with other PNH-targeted agents is not considered approvable under this coverage guideline.

Use of Fabhalta (iptacopan) for off‑label or investigational indications is considered not approvable when supporting evidence is insufficient or not generally accepted by the medical community. The policy requires evidence consistent with CMS‑recognized compendia or acceptable peer‑reviewed literature. When evaluating investigational/off‑label requests, reviewers should consider whether the patient and disease characteristics are represented in the published evidence, whether the regimen studied matches the requested therapy, whether study outcomes are clinically meaningful (for example, ASCO‑recommended benchmarks such as a hazard ratio < 0.80 or survival benefits of at least 3 months for OS/PFS), and whether the study design is appropriate. Case reports and meeting abstracts without a full peer‑reviewed article generally do not provide adequate support.

Regimen	Indication	Coverage status	Source / rationale
Iptacopan (Fabhalta) monotherapy, oral dosing per FDA prescribing information and referenced clinical trials	Treatment of adult members with paroxysmal nocturnal hemoglobinuria (PNH)	Covered	Policy permits use for adult PNH per prescribing information and referenced trials; listed as covered indication in policy documentation

Billing and Coding

Applicable HCPCS/J-codesHCPCSCovered

J8999

iptacopan

Single dose limit — 200 mg

Single dose limit200 mg

SourceExclusion criterion: dosing exceeds single dose limit of 200 mg

Applies toAll Fabhalta (iptacopan) prescriptions covered under this policy

Maximum monthly quantity — 60 capsules (200 mg)

Maximum monthly quantity60 capsules (200 mg)

SourceExclusion criterion: treatment exceeds the maximum limit of 60 (200 mg) capsules/month

Implication

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization and Continuation Rules

Prior authorization is required for initiation and continuation of Fabhalta (iptacopan). Continuation (renewal) requests will be considered only if the member has not experienced disease progression on Fabhalta, the prior authorization was active within the last year with no lapse greater than 30 days, and no additional medications are being added to the continuation request.

Prior authorization required for all new starts
Continuation allowed when: no disease progression; authorization used within last 12 months with ≤30-day lapse; no additional meds added

Denial Risk

Concurrent Therapy Restriction

Fabhalta must not be used concurrently with other therapies indicated for paroxysmal nocturnal hemoglobinuria (PNH) such as eculizumab or ravulizumab. Concurrent use will result in denial of the request.

Concurrent administration with eculizumab or ravulizumab: EXCLUDED

Definitions and Safety Requirements

Risk Evaluation and Mitigation Strategy (REMS)

REMS requirementFABHALTA REMS required due to increased risk of serious infections from encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis, Haemophilus influenzae type b)

Vaccination requirementComplete or update vaccination for encapsulated bacteria at least 2 weeks prior to first dose unless delaying therapy poses greater risk

MonitoringMonitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected

Access restrictionIptacopan is available only through the restricted FABHALTA REMS program

Background

Fabhalta (iptacopan) is an oral complement inhibitor indicated for adults with paroxysmal nocturnal hemoglobinuria (PNH). Complement inhibition with agents such as iptacopan increases the risk of serious infections from encapsulated bacteria; accordingly, vaccination and REMS‑related safety steps are required prior to initiation. Treatment with Fabhalta is provided as monotherapy for PNH per the prescribing information and referenced clinical trials, and coverage is limited by the dosing thresholds specified in this policy.

Revision History

2025-06template_conversion_and_updatesLatest

Converted to the new Evolent guideline template; replaced prior policy UM ONC_1501; indication section and exclusion criteria were updated and maximum dosage form quantities were added.

2024-06-01policy_effective_new

Policy created as a new Evolent guideline for Fabhalta (iptacopan).