CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Fabhalta (iptacopan) — Coverage Criteria for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Defines indications, coverage criteria, and requirements for authorization of iptacopan (Fabhalta) for treatment of adult members with PNH and describes documentation and program requirements for safe use.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyFabhalta (iptacopan) — Coverage Criteria for PNH

Policy CodePolicy N/A

Change TypeTemplate conversiondosage limits added

Effective DateJune 2025

Next Review DateN/A

Key ActionObtain prior authorization (J8999) with documentation supporting FDA labeling/compendia evidence, vaccination status, and REMS considerations.

SourceLink

POLICY UPDATE CHANGES

Converted to new Evolent guideline template and updated indication section.

Added maximum dosage form quantities in exclusion criteria (treatment exceeds the maximum limit of 60 (200 mg) capsules/month).

60/monthmaximum treatment limit specified

200 mgsingle dose maximum

Jun 2025last revised date

J8999billing code listed

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Initial Therapy

Covered when ALL of the following are met

PNH indication and evidence: Adult member with paroxysmal nocturnal hemoglobinuria (PNH) and use supported by FDA labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or acceptable peer-reviewed literature as defined by the CMS Medicare Benefit Policy Manual Chapter 15.

References include FDA labeling and compendia; see policy references.

Safety and program requirements: Documentation of vaccination for encapsulated bacteria per ACIP at least 2 weeks prior to the first dose unless risks of delaying therapy outweigh risk of infection; enrollment/consideration of FABHALTA REMS due to increased risk of serious infections from encapsulated bacteria.

Monitor patients for early signs of invasive encapsulated bacterial infection.

The following are exclusions and usage limits for Fabhalta (iptacopan). Therapy is excluded if there is disease progression while taking iptacopan or if the member is receiving concurrent therapy with other PNH treatments (for example, eculizumab or ravulizumab). Dosing limits are enforced: a single dose must not exceed 200 mg, and treatment is limited to a maximum of 60 (200 mg) capsules per month. Requests exceeding these dosing thresholds will be denied.

Requests will also be excluded when Fabhalta is requested for investigational or off‑label indications that lack sufficient evidence or are not generally accepted by the medical community; see investigational/off‑label policy language for evidence requirements. These exclusion rules should be confirmed in submitted documentation and are grounds for denial when not met.

Use of Fabhalta (iptacopan) for investigational or off‑label indications is considered not medically necessary when the requested use is not supported by sufficient evidence or is not generally accepted by the medical community. Sufficient evidence is defined by consideration of whether the patient and disease characteristics are represented in the published evidence, whether the regimen is adequately represented, and whether reported study outcomes are clinically meaningful.

Acceptable evidence excludes uncontrolled case reports and abstracts without a full peer‑reviewed article. Nonrandomized trials with a significant number of subjects may provide supportive evidence, but overall coverage for off‑label uses requires documentation that meets the standards described here; uses failing to meet these criteria will be denied as investigational.

Regimen	Dose limits	Monthly limit	Coverage status
Iptacopan (Fabhalta) — oral monotherapy for adult PNH where coverage criteria met and exclusions absent	Single dose ≤ 200 mg	Maximum 60 capsules per month (200 mg strength)	Covered

Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Fabhalta (iptacopan, HCPCS J8999). Decisions will be based on FDA-approved labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or peer-reviewed literature that meets CMS standards. Evolent (on behalf of Neighborhood Health Plan of Rhode Island) processes all medication requests; medications not authorized may be deemed not approvable and not reimbursable.

Affected code: J8999 (Fabhalta, iptacopan) — prior authorization required
Clinical support must reference: FDA labeling; CMS-recognized compendia; NCCN or ASCO clinical guidelines; or peer-reviewed literature meeting CMS manual Chapter 15 standards

Denial Risk

Concurrent Therapy Prohibition

Concurrent use of Fabhalta with other PNH-targeted complement inhibitors is not permitted. Document absence of concurrent therapy in the request; requests indicating simultaneous treatment with excluded agents will be denied.

Concurrent therapy exclusion: eculizumab, ravulizumab, or other PNH therapies — must be absent
Requester must attest and document that the member is not receiving any other PNH complement inhibitor

Documentation Required

Required Supporting Documentation and Safety Measures

Supporting documentation must be provided with the authorization request. Acceptable sources include FDA-approved product labeling, CMS-recognized compendia, NCCN or ASCO clinical guidelines, or peer-reviewed literature that satisfies CMS Medicare Benefit Policy Manual Chapter 15 requirements. Documentation should address applicability of the evidence to the member (disease characteristics, regimen, and clinically meaningful outcomes).

Provide copies or citations of relevant sections from FDA labeling, CMS compendia, NCCN/ASCO guidelines, or peer-reviewed studies
If relying on peer-reviewed literature, include details showing patient population, regimen representation, outcomes (e.g., OS/PFS) and study design sufficient per CMS standards
Ensure REMS and vaccination requirements for encapsulated bacteria are addressed per labeling prior to initiation

Denial Risk

Triggers for Denial

Requests will be denied when exclusion criteria are met, including disease progression while receiving Fabhalta or evidence of concurrent use with other PNH therapies. Overdose (single dose >200 mg) or exceeding the monthly maximum (more than sixty 200 mg capsules per month) are additional denial triggers. Investigational or off‑label uses lacking adequate supporting evidence per CMS compendia or acceptable peer‑reviewed literature will be denied.

Denial triggers: documented disease progression on Fabhalta
Denial triggers: concurrent use with eculizumab, ravulizumab, or other PNH therapies
Denial triggers: dosing exceeds single dose limit of 200 mg or >60 (200 mg) capsules per month
Denial triggers: investigational/off-label indications without sufficient evidence as defined by CMS compendia or acceptable peer‑reviewed literature
Safety requirement: REMS enrollment and appropriate vaccinations for encapsulated bacteria per FDA labeling must be documented

Summary	Single-dose maximum	Monthly capsule maximum
Iptacopan (Fabhalta) oral dosing regimen for covered monotherapy in PNH	≤ 200 mg per single dose	≤ 60 capsules per month (200 mg capsules)

Definitions and Program Terms

inv-12: PNH — definition of Paroxysmal Nocturnal Hemoglobinuria and indication context

DefinitionParoxysmal Nocturnal Hemoglobinuria (PNH) is a hematologic disorder for which Fabhalta (iptacopan) may be used to treat adult members.

Indication contextPolicy covers iptacopan for adult patients with PNH when use is supported by FDA labeling or accepted compendia/peer‑reviewed evidence.

Safety considerationsAs a complement inhibitor, iptacopan increases risk of serious infections from encapsulated bacteria; vaccination per ACIP and REMS enrollment are required prior to therapy when possible.

inv-13: REMS — Risk Evaluation and Mitigation Strategy description for iptacopan

REMS program nameFABHALTA REMS (Risk Evaluation and Mitigation Strategy)

Reason for REMSIptacopan increases risk of serious infections caused by encapsulated bacteria (eg, S. pneumoniae, N. meningitidis, H. influenzae type b).

REMS requirementsIptacopan is available only through the restricted FABHALTA REMS program; ensure vaccination for encapsulated bacteria at least 2 weeks prior to first dose when possible and monitor for invasive disease.

Background

Iptacopan (Fabhalta) is an oral complement inhibitor indicated for treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). As a complement pathway inhibitor, Fabhalta increases the risk of serious infections from encapsulated bacteria; vaccination and REMS program considerations are required per the prescribing information and safety guidance.

When considering Fabhalta for PNH, ensure the indication is consistent with FDA labeling and that safety measures (including appropriate vaccination for encapsulated bacteria and REMS enrollment as applicable) are documented before initiating therapy.

OpenPayer

Fabhalta (iptacopan) — Coverage Criteria for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Coverage Criteria for Fabhalta (iptacopan)

Billing and Coding

Authorization, Documentation, and Provider Requirements

Approved Regimens and Limits

Line of Therapy Information

Definitions and Program Terms

Background

Revision History