CurrentCignaPolicy IP0307

Uridine triacetate (Xuriden) for hereditary orotic aciduria

Policy governs medical necessity review and coverage criteria for oral uridine triacetate (Xuriden) in the treatment of hereditary orotic aciduria for Cigna-administered health benefit plans.

Policy Summary

PayerCigna

PolicyUridine triacetate (Xuriden) for hereditary orotic aciduria

Policy CodePolicy IP0307

Change TypeMaterial addition to diagnostic criterianon‑material heading update

Effective Date12/15/2024

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation confirming diagnosis (molecular testing or specified clinical criteria including family history) and prescriber specialty.

SourceLink

POLICY UPDATE CHANGES

Added requirement: first-degree family relative (parent or sibling) with hereditary orotic aciduria as supportive clinical evidence.

Updated the condition heading from 'Hereditary Orotic Aciduria' to 'Hereditary Orotic Aciduria (Orotic Aciduria Type 1)'.

up to 12 moInitial approval duration

up to 12 moReauth approval duration

≈20Reported cases

<1:1,000,000Estimated prevalence

Coverage Criteria for Uridine Triacetate (Xuriden)

Initial Medical Necessity Criteria

Covered when ALL of the following are met for Hereditary Orotic Aciduria (Orotic Aciduria Type 1):

Overall: All criteria A and B must be met

A. Diagnostic confirmation: Diagnosis confirmed by ONE of the following: (i) molecular genetic testing confirming biallelic pathogenic variants in the UMPS gene OR (ii) clinical diagnosis supported by ALL of the specified clinical findings

A.ii Clinical diagnosis supported by: Clinical manifestations consistent with orotic aciduria (for example, megaloblastic anemia, leukopenia, neutropenia, failure to thrive, intellectual disability) AND first-degree family relative (parent or sibling) with hereditary orotic aciduria AND urinary orotic acid level above the normal reference range for the reporting laboratory

These three elements are listed as supporting criteria when molecular testing is not available

Policy Summary

PayerCigna

PolicyUridine triacetate (Xuriden) for hereditary orotic aciduria

Policy CodePolicy IP0307

Change TypeMaterial addition to diagnostic criterianon‑material heading update

Effective Date12/15/2024

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation confirming diagnosis (molecular testing or specified clinical criteria including family history) and prescriber specialty.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

B. Prescriber: Medication is prescribed by, or in consultation with, a metabolic specialist, geneticist, or physician specializing in the condition being treated

Reauthorization Criteria

Reauthorization covered when ALL of the following are met:

Continuation: Initial medical necessity criteria continue to be met AND documentation of beneficial response (for example, improved hematologic parameters, reduced urinary orotic acid and orotidine levels, improved growth)

Any use of uridine triacetate (Xuriden) for indications other than those specified in this policy is considered experimental, investigational, or unproven. Such uses are not supported by the evidence criteria in this policy and may be denied; criteria will be updated as new published data become available.

Per this policy, use of uridine triacetate beyond treatment of hereditary orotic aciduria (Orotic Aciduria Type 1) is not medically necessary. Requests for other indications should be considered noncovered unless and until supportive clinical evidence and policy criteria are established.

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial and reauthorization approvals are up to 12 months.

Initial approval duration: up to 12 months
Reauthorization approval duration: up to 12 months

Documentation Required

Reauthorization Requirement

Continuation (reauthorization) requires that the initial medical necessity criteria continue to be met and that there is documentation of a beneficial response to therapy.

Documentation of beneficial response examples: improved hematologic parameters, reduced urinary orotic acid and orotidine levels, improved growth
Prescriber: medication must be prescribed by, or in consultation with, a metabolic specialist, geneticist, or physician specializing in the condition being treated

Denial Risk

Non-covered Uses

Use for any indication other than Hereditary Orotic Aciduria is considered experimental, investigational, or unproven and may be denied.

Non-covered: any diagnosis or indication other than Hereditary Orotic Aciduria (Orotic Aciduria Type 1)

Background

Hereditary orotic aciduria (Orotic Aciduria Type 1) is an extremely rare autosomal recessive disorder caused by pathogenic variants in the UMPS gene leading to deficient UMP synthase, excess orotic acid, and clinical manifestations such as megaloblastic anemia, failure to thrive, and developmental delay. Estimated prevalence is less than 1:1,000,000 with approximately ≈20 reported cases in the literature. Xuriden (uridine triacetate) provides replacement uridine to reduce orotic acid overproduction and address biochemical and clinical features of the disorder.

Definitions

Hereditary Orotic Aciduria (Orotic Aciduria Type 1) — overview

DefinitionAn extremely rare autosomal recessive disorder of pyrimidine metabolism (also called orotic aciduria type 1) due to pathogenic variants in the UMPS gene leading to deficient UMP synthase and excess orotic acid.

Key biochemical defectDeficiency of uridine 5'-monophosphate (UMP) synthase prevents the final steps of pyrimidine biosynthesis, causing low uridine monophosphate and accumulation of orotic acid.

Typical clinical manifestationsAffected infants may develop megaloblastic anemia, developmental delays, failure to thrive, and urinary orotic acid crystals that can cause urinary obstruction.

DiagnosisMade by detailed patient and family history, clinical evaluation, urine examination for orotic acid; most diagnoses are confirmed by molecular genetic testing for UMPS variants (specialized laboratories).

Treatment rationaleUridine replacement (Xuriden) supplies circulating uridine and via feedback inhibition reduces overproduction of orotic acid.

EpidemiologyExtremely rare: estimated prevalence <1:1,000,000 live births with approximately 20 cases reported in the literature.