Elamipretide (Forzinity) coverage for Barth syndrome
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Defines medical necessity and prior authorization criteria for Forzinity (elamipretide) for members, primarily for treatment of Barth syndrome in eligible patients and guidance for other/off-label uses referencing related policies. Affects commercial, HIM, and Medicaid lines of business.
Drug is now FDA approved - criteria updated per FDA labeling; added weight requirement; added requirement for documentation of impaired muscle strength; added quantity limit of 1 vial per 7 days; in continued therapy, specified positive response to therapy.
Coverage Criteria for Forzinity (elamipretide)
Continuation Therapy
Covered when ALL of the following are met for continued therapy (Barth syndrome):
Approval duration: Medicaid/HIM 12 months; Commercial 6 months or to member renewal date, whichever is longer
Non‑FDA approved indications that are not specifically addressed in this policy are not authorized for coverage unless the request includes sufficient documentation of efficacy and safety in accordance with applicable off‑label use or evidence‑of‑coverage policies. Refer to the off‑label policies for each line of business (e.g., CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid) when evaluating non‑labeled uses.
When a requested use is not listed among authorized indications, route the request per the policy framework: if a recent label change (within the last 6 months) may apply, follow the no‑coverage or non‑formulary routing per line‑of‑business policies; otherwise route requests for unlisted uses to the applicable off‑label use policy (CP.CPA.09 commercial, HIM.PA.154 HIM, CP.PMN.53 Medicaid). Uses for non‑FDA approved indications lacking sufficient supporting evidence or adherence to these off‑label/formulary processes are considered not authorized.
Initial Authorization Criteria
Initial Therapy
Initial authorization criteria for Barth syndrome:
Initial approval duration: 6 months
Criteria for Continued Coverage
Continuation Therapy
Criteria for continued coverage in Barth syndrome:
Approval duration: Medicaid/HIM 12 months; Commercial 6 months or to member renewal date, whichever is longer
Coding and Dosing Key Facts
Provider Requirements and Administrative Actions
Prior authorization required with supporting documentation
Prior authorization is required. The request must include documentation that the member meets the approval criteria: confirmed TAZ gene mutation by DNA testing; prescribing clinician is one of the specified specialists or there is a documented consult; member weight ≥ 30 kg; documentation of impaired muscle strength; and dosing within limits (≤ 40 mg/day and ≤ 1 vial per 7 days). HCPCS codes referenced for billing are C9399 and J3490.
- Diagnosis confirmed by DNA testing for a TAZ mutation
- Prescribed by or in consultation with an approved specialist (clinical geneticist, metabolic disease specialist, endocrinologist, cardiologist, hematologist, or neurologist)
- Weight ≥ 30 kg
- Documentation of impaired muscle strength (e.g., knee extensor strength by handheld dynamometry)
- Dose limits: ≤ 40 mg per day and ≤ 1 vial per 7 days
Follow no-coverage/non‑formulary or off‑label routing for unlisted uses
If the requested use is not listed under the policy's authorized indications (or if a recent label change is not yet reflected), route the request per the referenced no-coverage/non‑formulary or off-label use policies for the applicable line of business rather than through this policy.
- Refer to no coverage/non‑formulary policies when drug is on or not on formulary (CP.CPA.190, HIM.PA.33, CP.PMN.255 and HIM.PA.103, CP.PMN.16 as applicable)
- Refer to off‑label use policy when the use is not listed and recent label change guidance does not apply (CP.CPA.09, HIM.PA.154, CP.PMN.53)
Submit office notes, labs, and clinical measures to support criteria
Provider must submit clinical documentation (office chart notes, lab results, or other clinical information) that demonstrates the member meets all approval criteria, including DNA testing confirming a TAZ mutation, weight documentation showing ≥ 30 kg, and objective measures of impaired muscle strength.
- Office chart notes or consult documenting specialist involvement
- DNA test results confirming TAZ gene mutation
- Weight documentation (e.g., charted weight) showing ≥ 30 kg
- Objective measure of impaired muscle strength (e.g., knee extensor strength by handheld dynamometry)
Denial risk if required documentation is not provided
Requests missing required documentation may be denied. Examples include absence of DNA confirmation of a TAZ mutation, lack of documented weight meeting the ≥ 30 kg requirement, or missing objective documentation of impaired muscle strength. Non‑FDA uses without adequate off‑label/formulary documentation may also be denied.
- Missing DNA test confirming TAZ mutation may lead to denial
- No documented weight ≥ 30 kg may lead to denial
- Lack of objective documentation of impaired muscle strength may lead to denial
- Non‑FDA approved indications without evidence per off‑label or no‑coverage policies may be denied
Step Therapy and Related Routing
| Scenario | Routing / Policy to follow |
|---|---|
| Recent label change within last 6 months not yet reflected in this policy (e.g., new indication, age expansion, or dosing regimen) | Refer to applicable no-coverage criteria policy (for drugs on formulary/PDL) or non-formulary policy (for drugs not on formulary/PDL) for the relevant line of business: CP.CPA.190 (Commercial), HIM.PA.33 (HIM) or CP.PMN.255 (Medicaid) for no-coverage; or CP.CPA.190 (Commercial), HIM.PA.103 (HIM) or CP.PMN.16 (Medicaid) for non-formulary routing. |
| Requested use not specifically listed in authorized indications and no recent label change applies | Refer to the off‑label use policy for the relevant line of business: CP.CPA.09 (Commercial), HIM.PA.154 (HIM) or CP.PMN.53 (Medicaid). |
Quantity Limits and Dosing Units
Site of Care and Administration
Subcutaneous administration per dosing regimen (home allowed)
Forzinity is administered subcutaneously per the dosing regimen (Barth syndrome dosing: 40 mg SC once daily); the policy does not impose additional site‑of‑care restrictions beyond SC administration at home.
- Barth syndrome dosing regimen: 40 mg SC QD; maximum 40 mg/day.
- Site of care: home SC administration per dosing regimen; no specific site‑of‑care restrictions specified in the policy.
Background and Drug Information
Forzinity (elamipretide) is a mitochondrial‑targeting peptide that binds cardiolipin. It received FDA accelerated approval to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg, based on improvement in knee extensor muscle strength as an intermediate endpoint.
Definitions
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