CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Rydapt (midostaurin) — Drug Coverage / Utilization Management

Defines accepted indications, inclusion and exclusion criteria, and utilization management procedures for coverage of Rydapt (midostaurin) for members; applies to medication requests processed by the UM department and network ordering providers.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyRydapt (midostaurin) — Drug Coverage / Utilization Management

Policy CodePolicy N/A

Change TypeNo material change

Effective DateMay 31, 2024

Next Review DateMay 10, 2023

Key ActionObtain prior authorization; continuation requests may be approved if medication was used within the last year and there is no disease progression or intolerance.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

FLT3-mutant AMLdocumented indication

ASM / SM-AHN / MCLsystemic mastocytosis indications

120/moAML dosing cap

240/moASM/MCL/SM-AHN dosing cap

50 mg / 100 mgsingle dose max

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Coverage Criteria for Rydapt (midostaurin)

Initial and Continuation Therapy Criteria

Covered when ALL of the following are met for the specified indication

Continuation Requests: If request is for continuation: requested medication was used within the last year AND member has not experienced disease progression or intolerance to the requested medication AND no additional medication(s) are being added to the continuation request

applies to continuation requests only

AML Indication: Member has documented FLT3 mutation-positive AML (ITD and/or TKD) detected by an FDA-approved test (e.g., LeukoStrat CDx) AND Rydapt (midostaurin) will be used for induction in combination with chemotherapy OR for post-remission/consolidation therapy with or without chemotherapy following complete response to prior induction therapy

Systemic Mastocytosis Indication: Member has aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL) AND Rydapt (midostaurin) will be used as a single agent

Exclusions for Rydapt (midostaurin) include: disease progression on Rydapt or another FLT3 inhibitor (e.g., gilteritinib), dosing that exceeds single-dose limits (the policy specifies a single-dose maximum of 50 mg for AML and 100 mg for ASM, MCL, or SM-AHN), absence of a documented FLT3 mutation for AML, and requests that would exceed the monthly capsule limits (120 × 25 mg capsules/month for AML; 240 × 25 mg capsules/month for ASM/MCL/SM-AHN).

The policy also excludes investigational or off-label uses when the indication is not supported by sufficient evidence or is not generally accepted by the medical community; specific insufficiency criteria include lack of representative patient/ disease characteristics, regimen mismatch with published evidence, absence of clinically meaningful outcomes (per ASCO-recommended thresholds), inadequate experimental design for the question, overreliance on uncontrolled case reports, and reliance on abstracts without full peer‑reviewed articles.

Off‑label or investigational uses of Rydapt are considered not approvable when the supporting evidence is inadequate. The policy describes the evidence insufficiency criteria that will result in investigational determinations: inadequate representation of the patient and cancer in published studies; the administered regimen in practice not matching regimens studied; reported outcomes that are not clinically meaningful (ASCO benchmarks such as Hazard Ratio < 0.80 or survival benefit thresholds are referenced); experimental designs that cannot appropriately address the clinical question; reliance solely on uncontrolled case reports; and dependence on meeting abstracts without full peer‑reviewed publications. Such gaps in evidence or acceptance by recognized compendia will render off‑label indications investigational and not approvable.

This investigational determination follows the exclusion framework and is intended to ensure coverage decisions rely on CMS‑recognized compendia, FDA labeling, NCCN/ASCO guidance, or acceptable peer‑reviewed literature rather than limited or anecdotal reports.

Initial Therapy Coverage Criteria

Initial Therapy

Initial therapy coverage criteria per indication:

AML initial therapy: Documented FLT3 mutation-positive AML (ITD and/or TKD) detected by an FDA-approved test (e.g., LeukoStrat CDx) AND planned use of Rydapt (midostaurin) in combination with induction chemotherapy (or for post-remission/consolidation therapy following complete response)

ASM/MCL/SM-AHN initial therapy: Diagnosis of aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL) AND planned use of Rydapt (midostaurin) as a single agent

Continuation Therapy Criteria

Continuation Therapy

Continuation requests may be approved without meeting full policy if all continuation conditions are met:

Continuation conditions: Requested medication was used within the last year AND the member has not experienced disease progression or intolerance to the requested medication AND no additional medication(s) are being added to the continuation request

Coding and Quantity Limits

Monthly capsule limits — Rydapt (midostaurin)

AML monthly capsule limit120 (25 mg) capsules per month

ASM / MCL / SM-AHN monthly capsule limit240 (25 mg) capsules per month

Unit strength25 mg capsule

Single dose limits — Rydapt (midostaurin)

Single dose limit — AML50 mg single dose

Single dose limit — ASM / MCL / SM-AHN100 mg single dose

ContextExceeding these single dose limits is listed as an exclusion criterion

Provider Actions, Prior Authorization & Documentation

Prior Authorization

Prior Authorization Required

Prior authorization required. Continuation requests are exempt when the medication was used within the last year, there is no documented disease progression or intolerance, and no additional medications are being added to the continuation request.

Continuation exemption: medication used within last year; no disease progression or intolerance; no additional meds added.

Documentation Required

Required Supporting Documentation

Requests must be supported by one of the following sources: FDA-approved product labeling, CMS-recognized compendia, NCCN or ASCO clinical guidelines, or acceptable peer-reviewed literature that meets CMS standards.

Acceptable sources: FDA labeling; CMS-recognized compendia; NCCN or ASCO guidelines; peer-reviewed literature meeting CMS Medicare Benefit Policy Manual Chapter 15 requirements.

Definitions and Indication Labels

FLT3 mutation-positive AML — definition and testing context

DefinitionFLT3 mutation-positive AML: AML with FLT3 internal tandem duplication (ITD) and/or tyrosine kinase domain (TKD) mutations detected by an FDA-approved test (e.g., LeukoStrat CDx)

Testing requirementMutation must be detected by an FDA-approved test for coverage consideration

Coverage contextRequired for Rydapt use in induction or post-remission/consolidation therapy (with or without chemotherapy)

Systemic mastocytosis subtypes — definitions

ASMAggressive systemic mastocytosis — covered indication when Rydapt is used as single agent

SM-AHNSystemic mastocytosis with associated hematological neoplasm — covered indication when Rydapt is used as single agent

Background

Midostaurin (Rydapt) is a targeted tyrosine kinase inhibitor used in oncology. It is indicated for FLT3‑mutation positive acute myeloid leukemia (AML) as part of induction and post‑remission/consolidation therapy in combination with chemotherapy, and as a single agent for advanced systemic mastocytosis subtypes including aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM‑AHN), and mast cell leukemia (MCL). Usage and monitoring should align with the FDA label and accepted compendia or guidelines (NCCN/ASCO) as cited by the policy.

Note: refer to the policy for utilization management requirements (prior authorization, continuation rules, required supporting documentation) and dosing quantity limits when evaluating requests.

Quantity Limits Summary

Rydapt (midostaurin) — monthly quantity limits (summary)

AML quantity limit (actionable)120 capsules/month (25 mg) for FLT3-mutant AML

ASM / MCL / SM-AHN quantity limit240 capsules/month (25 mg) for ASM, SM-AHN, or MCL

Capsule strength25 mg capsules — limits expressed in capsule count per month

Site of Care / Administration Notes

Note

Follow FDA label for medication management; UM department processes requests

Refer to the FDA label/package insert for medication administration, monitoring, and other management details. All medication requests for Rydapt are processed by the UM department.