CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Pemazyre (pemigatinib) — Coverage Criteria

Defines criteria for authorization, continuation, and exclusion for pemigatinib (Pemazyre) for cholangiocarcinoma and myeloid/lymphoid neoplasms for members whose medication requests are processed by the payer's UM department.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyPemazyre (pemigatinib) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJul 26, 2024

Next Review DateN/A

Key ActionObtain prior authorization with documentation of indication, prior therapies, and confirmatory FGFR testing.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2covered indications

13.5 mgsingle dose cap

42/28/14max tablets/mo (cholangiocarcinoma)

90/60/30max tablets/mo (MLNs)

UM Deptapproval authority

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inv-01: Cholangiocarcinoma - Initial Therapy

Covered when ALL of the following are met:

Cholangiocarcinoma coverage criteria: The member has unresectable or metastatic cholangiocarcinoma with disease progression on or after one prior therapy; AND a positive test for FGFR2 (fibroblast growth factor receptor 2) gene fusion or rearrangement confirmed in the tumor by an approved test (e.g., FoundationOne CDx or other gene sequencing test).

Pemigatinib used as single-agent therapy.

inv-02: Myeloid/Lymphoid Neoplasms - Initial/Relapsed Therapy

Covered when ALL of the following are met:

MLN coverage criteria: The member has relapsed after stem cell transplantation and/or after disease-modifying therapies (e.g., chemotherapy) for myeloid/lymphoid neoplasms; AND the tumor is positive for FGFR1 (fibroblast growth factor receptor 1) rearrangement.

Pemigatinib may be used as monotherapy.

Coverage is not supported when any of the exclusion conditions below apply. Deny or non‑authorize requests for pemigatinib if there is disease progression while receiving pemigatinib or if the member has experienced progression on a prior FGFR2 inhibitor (for example, futibatinib/Lytgobi). A request lacking a confirmatory tumor test demonstrating an FGFR2 fusion/rearrangement (cholangiocarcinoma) or an FGFR1 rearrangement (myeloid/lymphoid neoplasms) should be excluded.

Dosing that exceeds the policy limits is an exclusion: a single dose greater than 13.5 mg is not allowed. For cholangiocarcinoma, requests exceeding the monthly tablet limits of 42 tablets (4.5 mg), 28 tablets (9 mg), or 14 tablets (13.5 mg) must be excluded. For myeloid/lymphoid neoplasms, requests exceeding the monthly tablet limits of 90 tablets (4.5 mg), 60 tablets (9 mg), or 30 tablets (13.5 mg) must be excluded.

Investigational or off‑label use is excluded when the indication is not supported by sufficient evidence in CMS‑recognized compendia or acceptable peer‑reviewed literature. The policy does not consider case reports or meeting abstracts without full peer‑reviewed articles to provide adequate supportive evidence; requests for indications lacking robust clinical evidence (appropriate patient representation, relevant regimens, clinically meaningful outcomes, and sound study design) should be denied.

Pemigatinib use for off‑label indications is considered not medically necessary when the requested indication is not supported by CMS‑recognized compendia or acceptable peer‑reviewed literature. The policy requires adequate evidence that the published studies appropriately represent the patient population and treatment regimen, report clinically meaningful outcomes, and use study designs sufficient to answer the clinical question.

Case reports are generally regarded as uncontrolled anecdotal evidence and do not meet the evidentiary threshold. Abstracts or meeting presentations without a corresponding full peer‑reviewed article also lack sufficient evidence to support coverage for off‑label uses.

Initial Therapy — Authorization Requirements

inv-12: Initial Therapy Criteria

Initial use covered when ALL indication-specific molecular and prior therapy criteria below are met:

Cholangiocarcinoma initial: Unresectable or metastatic cholangiocarcinoma with disease progression on or after one prior therapy; AND tumor-confirmed FGFR2 fusion or rearrangement by an approved test (e.g., FoundationOne CDx).

Single-agent pemigatinib.

Myeloid/Lymphoid Neoplasms initial/relapsed: Relapse after stem cell transplantation and/or after disease-modifying therapies for MLNs; AND tumor/disease positivity for FGFR1 rearrangement confirmed by an appropriate test.

Pemigatinib as monotherapy.

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Requests must include documentation of the indication, prior therapies, and confirmatory FGFR testing results; requests will be reviewed by the UM department.

All requests require prior authorization review by the UM department.
Include prior therapy history and evidence of disease progression where applicable.
Attach copy of confirmatory tumor testing (e.g., FoundationOne CDx or equivalent).

Step Therapy

Line‑of‑Therapy Requirement

For cholangiocarcinoma, pemigatinib is supported only after disease progression on or after one prior systemic therapy. Ensure documentation clearly shows prior treatment and progression date.

Applicable diagnosis: unresectable or metastatic cholangiocarcinoma.
Must document at least one prior therapy and subsequent disease progression prior to initiating pemigatinib.

Documentation Required

Required Documentation

Provide complete supporting documentation including prior therapy history, evidence of disease progression, and confirmatory tumor testing showing the relevant FGFR gene fusion/rearrangement. Missing documentation may delay or result in denial.

Required: pathology or molecular diagnostic report confirming FGFR2 fusion/rearrangement for cholangiocarcinoma or FGFR1 rearrangement for MLNs (e.g., FoundationOne CDx or equivalent).
Required: prior systemic therapy records and documentation of progression (dates, regimen names).
Include prior authorization form, relevant clinic notes, and radiology/pathology reports.

Denial Risk

Triggers for Denial

Requests meeting any exclusion criteria should be denied. Common triggers for denial include disease progression on pemigatinib or prior FGFR2 inhibitor therapy, absence of confirmatory FGFR testing, dosing or quantity limits exceeded, and investigational/off‑label uses lacking sufficient evidence.

Deny if the member had disease progression while receiving pemigatinib or progressed on a prior FGFR2 inhibitor (e.g., futibatinib).
Deny if no confirmatory test is available to document the required FGFR2 (cholangiocarcinoma) or FGFR1 (MLNs) fusion/rearrangement.
Deny if requested dosing exceeds single dose limit of 13.5 mg or monthly tablet limits (Cholangiocarcinoma: 42x4.5 mg, 28x9 mg, 14x13.5 mg; MLNs: 90x4.5 mg, 60x9 mg, 30x13.5 mg).
Deny investigational or off‑label uses that lack sufficient supporting evidence per listed criteria (not supported by CMS compendia or acceptable peer‑reviewed literature).

Key Definitions

FGFR2 fusion/rearrangement (cholangiocarcinoma)

DefinitionTumor-confirmed FGFR2 (fibroblast growth factor receptor 2) gene fusion or rearrangement detected in the tumor by an approved test (e.g., FoundationOne CDx or another gene sequencing test)

Indication contextRequired for use of pemigatinib as single‑agent therapy in unresectable or metastatic cholangiocarcinoma

Test examplesApproved tests include FoundationOne CDx or other gene sequencing tests that confirm FGFR2 fusion/rearrangement in tumor cells

FGFR1 rearrangement (MLNs)

DefinitionTumor or disease positivity for FGFR1 (fibroblast growth factor receptor 1) rearrangement

Indication contextRequired for pemigatinib monotherapy in relapsed myeloid/lymphoid neoplasms after stem cell transplantation and/or disease‑modifying therapies

Clinical scenarioApplies when MLN has relapsed following transplant and/or chemotherapy and FGFR1 rearrangement is confirmed

Step	Requirement	Coverage status
1	Member has unresectable or metastatic cholangiocarcinoma with disease progression on or after one prior therapy	Prior therapy required before coverage
2	Tumor test confirms FGFR2 fusion or rearrangement by an approved test (e.g., FoundationOne CDx or other gene sequencing test)	Molecular confirmation required
Notes	Pemigatinib use as a single agent; both requirements must be met for coverage

Background

Pemigatinib (Pemazyre) is a targeted FGFR inhibitor indicated for tumors with relevant FGFR gene alterations. In cholangiocarcinoma, pemigatinib is used as single‑agent therapy for unresectable or metastatic cholangiocarcinoma with an FGFR2 fusion/rearrangement after disease progression on or after at least one prior therapy. In hematologic disease, pemigatinib may be used as monotherapy for certain myeloid/lymphoid neoplasms (MLNs) that have relapsed after stem cell transplantation and/or disease‑modifying therapies when the disease is positive for an FGFR1 rearrangement. Coverage requires confirmatory molecular testing (e.g., FoundationOne CDx or other sequencing) and adherence to the policy's prior‑therapy and dosing limits.

OpenPayer

Pemazyre (pemigatinib) — Coverage Criteria

Coverage Criteria for Pemazyre (pemigatinib)

Initial Therapy — Authorization Requirements

Provider Requirements and Prior Authorization

Reauthorization / Continuation Criteria

Key Definitions

Step Therapy Requirements

Quantity Limits and Dosing Constraints

Background