Subject: Carbaglu 2122-A SGM P2023
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CARBAGLU
(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.
A. FDA-Approved Indications
1. Acute hyperammonemia in patients with NAGS deficiency
Carbaglu is indicated as an adjunctive therapy in pediatric
and adult patients for the treatment of acute
hyperammonemia due to the deficiency of the hepatic
enzyme N-acetylglutamate synthase (NAGS). 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 in patients with NAGS
deficiency Carbaglu is indicated for maintenance therapy
in pediatric and adult patients for the treatment of
chronic hyperammonemia due to the deficiency of the
hepatic enzyme NAGS. 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 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 academia 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.
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.
June 18, 2023
