Subject: carglumic acid-Carbaglu 2122-A SGM P2024
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CARBAGLU
(carglumic acid)
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carglumic acid
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Policy:
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are
considered a covered benefit provided that all the approval criteria are met and the member
has no exclusions to the prescribed therapy.
FDA-Approved Indications
1. Acute Hyperammonemia due to N-acetylglutamate Synthase (NAGS) Deficiency
Carbaglu is indicated in pediatric and adult patients as adjunctive therapy to standard of
care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase
(NAGS) deficiency. During acute hyperammonemic episodes, concomitant administration of
Carbaglu with other ammonia lowering therapies, such as alternate pathway medications,
hemodialysis, and dietary protein restriction is recommended.
2. Chronic Hyperammonemia due to N-acetylglutamate Synthase (NAGS) Deficiency
Carbaglu is indicated in pediatric and adult patients as maintenance therapy for the
treatment of chronic hyperammonemia due to NAGS deficiency. During maintenance
therapy, the concomitant use of other ammonia lowering therapies and protein restriction
may be needed based on plasma ammonia levels.
3. Acute Hyperammonemia due to Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
Carbaglu is indicated in pediatric and adult patients as adjunctive therapy to standard of
care for the treatment of acute hyperammonemia due to PA or MMA.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review for
NAGS deficiency:
A. Initial requests:
1. Enzyme assay, biochemical or genetic testing results supporting diagnosis of NAGS
deficiency; and
2. Lab results documenting baseline plasma ammonia levels.
B. Continuation of therapy requests: lab results documenting a reduction in plasma ammonia
levels from baseline.
III. CRITERIA FOR INITIAL APPROVAL
A. N-acetylglutamate Synthase (NAGS) Deficiency
Authorization of 12 months may be granted for members with a diagnosis of NAGS
deficiency when both of the following criteria are met:
1. The diagnosis is confirmed by enzymatic, biochemical, or genetic testing.
2. The member has elevated plasma ammonia levels at baseline.
B. Methylmalonic Acidemia
Authorization of 12 months may be granted for members who have a diagnosis of
methylmalonic acidemia.
C. Propionic Acidemia
Authorization of 12 months may be granted for members who have a diagnosis of propionic
acidemia.
IV. CONTINUATION OF THERAPY
A. N-acetylglutamate Synthase (NAGS) Deficiency
Authorization of 12 months may be granted for continued treatment in members requesting
reauthorization for NAGS deficiency who are experiencing benefit from therapy as
evidenced by a decrease in ammonia levels from baseline.
B. Methylmalonic Acidemia or Propionic Acidemia
Authorization of 12 months may be granted for continued treatment in members requesting
reauthorization for methylmalonic acidemia or propionic acidemia who are experiencing
benefit from therapy as evidenced by disease stability or disease improvement.
Place of Service:
Outpatient
The above policy is based on the following references:
- Carbaglu [package insert]. Lebanon, NJ: Recordati Rare Diseases, Inc.; August 2021.
- Filippi L, Gozzini E, Fiorini P, et al. N-carbamylglutamate in emergency management of hyperammonemia in neonatal acute onset propionic and methylmalonic aciduria. Neonatology. 2010;97(3):286-290.
- Levrat V, Forest I, Fouilhoux A, et al. Carglumic acid: an additional therapy in the treatment of organic acidurias with hyperammonemia. Orphanet J Rare Dis. 2008;3:2.
- Gebhardt B, Vlaho S, Fischer D, et al. N-carbamylglutamate enhances ammonia detoxification in a patient with decompensated methylmalonic aciduria. Mol Genet Metab. 2003:79(4):303-304.
- Gebhardt B, Dittrich S, Parbel S, et al. N-carbamylglutamate protects patients with decompensated propionic aciduria from hyperammonaemia. J Interit Metab Dis. 2005;28(2):241-244.
- Schwahn BC, Pleterse L, Bisset WM, et al. Biochemical efficacy of N-carbamylglutamate in neonatal severe hyperammonaemia due to propionic acidaemia. Eur J Pediatr. 2010;169(1):133-134.
- Valayannopoulos V, Baruteau J, Delgado MB, et. al. Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study. Orphanet J Rare Dis. 2016;11:32.
- Baumgartner MR, Hörster F, Dionisi-Vici C, et. al. Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia. Orphanet J Rare Dis. 2014; 9:130.
- carglumic acid [package insert]. Deer Park, IL; Eton Pharmaceuticals, Inc.; March 2022.
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.
April 17, 2024
