Subject: Bile Acid Synthesis Disorder Agents

Drug

Cholbam®  (cholic acid)

Policy:

Note: The provision of physician samples does not guarantee coverage under the provisions of the pharmacy benefit. All criteria below must be met in order to obtain coverage of each of these medications.

1. Precertification Criteria

Under some plans, including those that use an open or closed formulary, cholic acid (Cholbam) is subject to precertification. If precertification requirements apply, Aetna considers this medication to be medically necessary for those members who meet the following precertification criteria:

 

Bile acid synthesis disorder (BASD)

1. The member has a documented diagnosis or a bile acid synthesis disorder due to a single enzyme defect (see Appendix 1).

 

Peroxisomal disorder (PD)

1. The member has a documented diagnosis of a peroxisomal disorder (see Appendix 2); and
2. The member exhibits manifestations of liver disease (e.g. jaundice, enlarged liver, abnormal liver enzyme tests), steatorrhea, or complications from decreased fat soluble vitamin absorption; and
3. Cholic acid (Cholbam) is being used as adjunctive treatment.

 

 

Special Notes:

 Appendix 1: Bile Acid Synthesis Disorder Subtypes

• 3-beta-hydroxy-delta-5-C27-steroid oxidoreductase (3β-HSD) deficiency [Congenital bile acid synthesis defect type 1]
• Alpha-methylacyl-CoA racemase (AMACR) deficiency [Congenital bile acid synthesis defect type 4]
• Amino acid n-acyltransferase deficiency
• Bile acid CoA ligase deficiency
• Cholesterol 7alpha-hydroxylase (CYP7A1) deficiency
• Delta4-3-oxosteroid 5-beta-reductase (AKR1D1) deficiency [Congenital bile acid synthesis defect type 2]
• Oxysterol 7-alpha-hydroxylase deficiency [Congenital bile acid synthesis defect type 3]
• Sterol 27-hydroxylase deficiency (cerebrotendinous xanthomatosis; CTX)
• Trihydroxycholestanoic acid CoA oxidase deficiency

 

 

Appendix 2: Peroxisomal Disorder Subtypes

• Zellweger syndrome (ZWS)
• Adrenoleukodystrophy (NALD)
• Infantile Refsum disease (IRD)
• Rhizomelic chondrodysplasia punctata type 1 (RCDP1)
• X-linked adrenoleukodystrophy (X-ALD)
• Refsum disease (phytanoyl CoA hydroxylase deficiency)
• Acyl CoA oxidase deficiency (pseudo-NALD)
• D-bifunctional protein deficiency (DBP deficiency)
• Rhizomelic chondrodysplasia punctata type 2 (RCDP2; dihydroxy-acetone phosphate acyltransferase deficiency)
• Alpha-methylacyl-CoA racemase deficiency (AMACR deficiency)
• Peroxisomal sterol carrier protein-X deficiency (SCPx deficiency)
• Acatalasemia (catalase deficiency)
• Hyperoxaluria type 1 (alanine glyoxylate aminotransferase deficiency)

 

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Cholbam prescribing information. Manchester Pharmaceuticals Inc. San Diego, CA. March 2015.

 

 

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

March 24, 2021