CurrentBlue Cross Blue Shield - IowaPolicy N/A

Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD)

Policy governs authorization criteria for Dojolvi (triheptanoin) as a source of calories/fatty acids for pediatric and adult patients with molecularly confirmed LC-FAOD, including required documentation, prescriber specialty, initial and continuation approval durations, and dosing/billing guidance.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyDojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD)

Policy CodePolicy N/A

Change TypeAdministrative revision (no material clinical change)

Effective DateJun 4, 2025

Next Review Date

Key ActionSubmit chart note documenting at least one hospitalization or ER visit within past year for rhabdomyolysis, cardiomyopathy, or hypoglycemia and chart/lab documentation of low enzyme activity in cultured fibroblasts and/or pathogenic mutations confirmed by genetic testing.

SourceLink

POLICY UPDATE CHANGES

Reviewed August 2025; revised April 2025; current effective date June 4, 2025.

6Covered LC-FAOD Diagnoses listed

6 moInitial approval duration

12 moContinuation approval duration

Coverage Summary

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Background: Dojolvi (triheptanoin) is FDA-approved as a source of calories and fatty acids for pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

Scope summary: Coverage is tied to documented diagnostic confirmation (biochemical or genetic), prior nutrition management including a low-fat/high-carbohydrate diet with medium-chain triglyceride (MCT) supplementation, evidence of recent serious clinical events (hospitalization or ER visit for rhabdomyolysis, cardiomyopathy, or hypoglycemia), prescription by or consultation with an enzyme/metabolic disorder specialist, and specified authorization durations (initial: 6 months; continuation: 12 months), with adherence to dosing limits per FDA labeling or guidelines.

Policy-level coverage summary

Covered diagnoses count6

Initial approval duration6 months

Continuation approval duration12 months

Authorization durations thresholdInitial: 6 months; Continuation: 12 months

Provider Actions & Requirements

Prior Authorization

Prior authorization required with documentation

Prior authorization required. Submit chart note documenting at least one hospitalization or ER visit within the past year for rhabdomyolysis, cardiomyopathy, or hypoglycemia, and include chart or laboratory documentation of low enzyme activity in cultured fibroblasts and/or genetic testing showing pathogenic mutation(s).

Documentation Required

Prescriber specialty requirement

Medication must be prescribed by or in consultation with a physician who specializes in the treatment of enzyme or metabolic disorders.

Documentation Required

Demonstrate benefit for continuation

For continuation requests, provide evidence of disease stability or improvement. Examples include improvement in cardiomyopathy, glycemic control, or exercise tolerance, or a reduction in episodes of cardiomyopathy, rhabdomyolysis, hypoglycemia, or hospitalizations.

Billing Rule

Dosing limits adherence

Approvals may be subject to dosing limits in accordance with FDA-approved labeling, accepted compendia, and/or evidence-based practice guidelines.

Background, Evidence & Definitions

Dojolvi is indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed LC-FAOD (FDA-approved indication).

This coverage policy ties authorization to specific clinical criteria: members must have one of the listed LC-FAOD diagnoses, have received a low-fat/high-carbohydrate diet with MCT supplementation, and have documentation of at least one hospitalization or ER visit within the past year for rhabdomyolysis, cardiomyopathy, or hypoglycemia. Diagnostic confirmation requires at least two diagnostic findings (e.g., disease-specific elevated acylcarnitines, low enzyme activity in cultured fibroblasts, and/or pathogenic mutations in the specified genes). Prescriptions must be by or in consultation with a metabolic/enzyme disorder specialist, and approvals are limited in duration with dosing limits per FDA labeling and evidence-based guidance.

Key evidence includes a Phase 2 single-arm 78-week study and multiple safety/efficacy reports (Vockley et al. 2017, 2019, 2021), as well as a randomized double-blind trial comparing triheptanoin with trioctanoin; these studies form the primary clinical support cited in the policy.

References enumerated in the policy include the Dojolvi package insert and peer-reviewed trials and extension studies: Vockley et al. (78-week Phase 2), Vockley et al. 2017 (24-week study), Vockley et al. 2021 (open-label extension), and the randomized triheptanoin versus trioctanoin trial (Gillingham et al.).

term	definition
LC-FAOD	Long-chain fatty acid oxidation disorders, including CPT1, CPT2, CACT, VLCAD, LCHAD, and TFP deficiencies.

Revision History

2025-08-01administrative

Reviewed August 2025; no material change to clinical policy statement.

2025-04-01administrative

Revised April 2025; change is administrative (no explicit clinical policy statement change).

2025-06-04administrativeLatest

Current effective date June 4, 2025 (policy in effect).