CurrentNeighborhood Health Plan of Rhode IslandPolicy 1817-A

Coverage criteria for Rydapt (midostaurin)

Coverage criteria for Rydapt (midostaurin) including FDA‑approved and compendial indications, prior authorization documentation, and authorization durations for members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCoverage criteria for Rydapt (midostaurin)

Policy CodePolicy 1817-A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionObtain prior authorization and submit medical record documentation of FLT3 mutation or FGFR1 rearrangement as applicable.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthstypical authorization duration

FLT3required biomarker for AML indication

ASM / SM-AHN / MCL / ISM / SSSMmastocytosis subtypes

Yescompendial uses included

Coverage Criteria for Midostaurin (Rydapt)

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Midostaurin (Rydapt) is not indicated as a single‑agent induction therapy for the treatment of patients with AML. For the FDA‑approved newly diagnosed FLT3‑mutated AML indication, midostaurin must be administered in combination with standard cytarabine and daunorubicin induction and with cytarabine consolidation; single‑agent use for induction in AML is outside the FDA indication and is not an approved use.

Uses of midostaurin that are not included in the FDA‑approved indications or listed compendial uses (for example, single‑agent AML induction) are considered outside the scope of coverage and may be denied as not medically necessary.

All indications for midostaurin that are not specified in the FDA‑approved labeling or the listed compendial uses are considered experimental/investigational and not medically necessary. Compendial uses that are accepted include relapsed/refractory AML (including post‑induction, reinduction, and maintenance), myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions, and systemic mastocytosis; other, unlisted indications will generally be denied.

Line of Therapy Specifications

first-line

Newly diagnosed AML (first‑line): Midostaurin is used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy for newly diagnosed FLT3 mutation‑positive AML (FLT3 mutation documented by an FDA‑approved test). Midostaurin is not indicated as single‑agent induction therapy.

Authorization may be granted for 12 months.

salvage

Salvage / other line uses: Compendial uses for AML including relapsed/refractory disease, post‑induction therapy, reinduction of residual disease, and maintenance therapy; and treatment of myeloid/lymphoid neoplasms with eosinophilia and FGFR1 or FLT3 rearrangement (chronic or blast phase) per compendia.

Documentation of rearrangement required for FGFR1/FLT3; authorization typically for 12 months.

Molecular Diagnostic Requirements

Denial Risk

Provider action: Non‑covered indications; Step requirement for ISM/SSSM

Provide medical record documentation of the specific molecular diagnostic result(s) with the prior authorization request. Lack of molecular diagnostic documentation may result in denial. For indications other than FLT3 mutation–positive AML (per FDA) or FGFR1‑rearranged myeloid/lymphoid neoplasms with eosinophilia, the request is considered non‑covered.

Non‑covered indications: any use not supported by documented FLT3 mutation (by FDA‑approved test) for newly diagnosed AML in combination with specified chemotherapy, or not documented FGFR1 rearrangement for myeloid/lymphoid neoplasms with eosinophilia.
Step requirement: for indolent systemic mastocytosis (ISM) or smoldering systemic mastocytosis (SSSM), ensure any plan‑required prior step therapies are completed and documented before authorization is requested.

Documentation Required

inv-06: Required molecular diagnostic documentation — medical record submission to initiate prior authorization

FL T3 — mutation‑positive as detected by an FDA‑approved test; FGFR1 — rearrangement documented in medical record. Required molecular diagnostic documentation must be submitted to initiate prior authorization review.

Regimens and Administration

Regimen	Indication	Coverage status
Midostaurin + cytarabine + daunorubicin (induction); midostaurin + cytarabine (consolidation)	Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation‑positive (as detected by an FDA‑approved test)	Covered when used in combination (not as single‑agent induction)

Regimen	Indication / positioning	Coverage status
Midostaurin single agent	Aggressive systemic mastocytosis (ASM); systemic mastocytosis with associated hematological neoplasm (SM‑AHN); mast cell leukemia (MCL); symptomatic indolent systemic mastocytosis (ISM) or smoldering systemic mastocytosis (SSSM) after first‑line therapy with a clinical trial or avapritinib	Covered

Provider Actions, Prior Authorization, and Non‑Covered Uses

Prior Authorization

Obtain prior authorization — typical approvals for 12 months

Prior authorization is required before therapy and approvals are typically granted for up to 12 months for covered indications including FLT3‑mutated AML (when not used as single‑agent induction), systemic mastocytosis subtypes, and neoplasms with FGFR1/FLT3 rearrangements.

Authorization of 12 months may be granted for FLT3 mutation‑positive AML when midostaurin is not used as single‑agent induction.
Authorization of 12 months may be granted for ASM, SM‑AHN, MCL, and for ISM/SSSM after required prior therapy.
Authorization of 12 months may be granted for myeloid/lymphoid neoplasms with FGFR1 or FLT3 rearrangement.

Step Therapy

Step requirement: single‑agent midostaurin for ISM/SSSM only after first‑line therapy

For symptomatic ISM or SSSM, single‑agent midostaurin may only be authorized after failure of first‑line therapy with a clinical trial or avapritinib.

Document prior first‑line therapy with a clinical trial or avapritinib in the medical record when requesting single‑agent midostaurin for ISM/SSSM.

Denial Risk

Risk of denial for non‑covered (experimental/investigational) uses

Use of midostaurin for indications not listed as FDA‑approved or in recognized compendia is considered experimental/investigational and is not medically necessary; such requests may be denied.

Requests for indications outside the FDA‑approved labeling and listed compendial uses should not be expected to meet medical necessity and are subject to denial.

Key Definitions

inv-09: FLT3 — definition and testing requirement

DefinitionFLT3 mutation: a molecular alteration detected by an FDA-approved test required to qualify for the FDA-approved midostaurin indication in newly diagnosed AML.

Testing requirementMutation must be identified using an FDA-approved test; the test result should be documented in the medical record for prior authorization review.

Therapy implicationFLT3 mutation-positive status supports use of midostaurin in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation (not as single-agent induction).

inv-10: Myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions — FGFR1/FLT3 rearrangement context

DefinitionMyeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions: neoplasms characterized by FGFR1 or FLT3 rearrangements for which midostaurin may be authorized per compendia.

DocumentationMedical record documentation of the FGFR1 (or FLT3) rearrangement is required to initiate prior authorization review.

Authorization noteCompendial use: authorization typically granted for 12 months for chronic or blast phase disease when rearrangement is documented.

Background

Midostaurin (Rydapt) is an oral kinase inhibitor with both FDA‑approved and compendial indications in hematologic malignancies. FDA approvals include use in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation for newly diagnosed FLT3‑mutated acute myeloid leukemia (AML), and single‑agent treatment for aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM‑AHN), and mast cell leukemia (MCL). Compendial uses include relapsed/refractory AML (including maintenance and other post‑induction settings), treatment of myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase gene fusions, and additional systemic mastocytosis indications.