CurrentCignaPolicy N/A

Metabolic Disorders - Betaine Anhydrous (Cystadane) Prior Authorization Policy

Covers prior authorization requirements and medical necessity criteria for betaine anhydrous powder (Cystadane and generic) for treatment of homocystinuria in Cigna-administered health benefit plans.

Policy Summary

PayerCigna

PolicyMetabolic Disorders - Betaine Anhydrous (Cystadane) Prior Authorization Policy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateSep 11, 2024

Key ActionObtain prior authorization and document genetic confirmation of a qualifying enzyme defect plus trial or concurrent vitamin B6/B12/folate supplementation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 yearapproval duration

3 genetic diagnosesqualifying diagnoses

2024-09-11policy review

No criteria changesrevision history

Coverage Criteria for Betaine Anhydrous (Cystadane)

Initial therapy (FDA‑approved indication)

Covered when ALL of the following are met

FDA-Approved Indication: A) Patient has a confirmed diagnosis based on genetic testing demonstrating one of the following: i. Cystathionine beta-synthase deficiency; OR ii. 5,10-methylenetetrahydrofolate reductase deficiency; OR iii. Cobalamin cofactor metabolism defect; AND B) Patient has tried or is concurrently receiving vitamin B6 (pyridoxine), vitamin B12 (cobalamin), or folate supplementation; AND C) The medication is prescribed by or in consultation with a geneticist, metabolic disease specialist, or a physician who specializes in the management of homocystinuria.

Approve for 1 year if all criteria met

Use of Cystadane (betaine anhydrous powder) is considered experimental, investigational, or unproven for any indication other than homocystinuria. Requests for coverage for non‑approved uses may be denied and will be reviewed only if new peer‑reviewed evidence supporting the use becomes available.

Provider Actions and Authorization Requirements

Policy Summary

PayerCigna

PolicyMetabolic Disorders - Betaine Anhydrous (Cystadane) Prior Authorization Policy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateSep 11, 2024

Key ActionObtain prior authorization and document genetic confirmation of a qualifying enzyme defect plus trial or concurrent vitamin B6/B12/folate supplementation.

SourceLink

On This Page

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Definitions

inv-08: Homocystinuria — definition of the disorder, genetic basis, clinical consequences

DefinitionA group of rare autosomal recessive disorders caused by mutations in enzymes that metabolize amino acids leading to elevated homocysteine levels.

Genetic basisCaused by mutations in specific enzymes involved in amino acid metabolism (autosomal recessive inheritance).

Representative enzyme defectsIncludes cystathionine beta‑synthase deficiency; 5,10‑methylenetetrahydrofolate reductase deficiency; and cobalamin cofactor metabolism defects.

Clinical consequencesElevated homocysteine can cause abnormalities in the central nervous system, eyes, skeletal system, and vascular system.

inv-09: Betaine anhydrous (Cystadane) — drug description and indication summary

Drug classMethylating agent (betaine anhydrous powder).

Brand and formulationCystadane (betaine anhydrous powder) and generic betaine anhydrous powder.

Indication summaryIndicated for treatment of homocystinuria to decrease elevated homocysteine blood concentrations in adults and pediatric patients.

Included homocystinuria typesIndicated for cystathionine beta‑synthase deficiency, 5,10‑methylenetetrahydrofolate reductase deficiency, and cobalamin cofactor metabolism defect.

Patient populationAdults and pediatric patients with homocystinuria.

Background

Betaine anhydrous (Cystadane) is a methylating agent indicated to reduce elevated plasma homocysteine concentrations in adults and pediatric patients with homocystinuria. Homocystinuria refers to a group of rare autosomal recessive disorders caused by mutations in enzymes involved in amino acid metabolism (for example, cystathionine beta‑synthase deficiency, 5,10‑methylenetetrahydrofolate reductase deficiency, and cobalamin cofactor metabolism defects) that can produce neurologic, ocular, skeletal, and vascular manifestations. Clinical evidence shows betaine lowers homocysteine and has been associated with improvements in seizures and cognitive/behavioral symptoms when used alongside vitamin therapy.