Carbaglu – NC Standard

• Carbaglu® (carglumic acid) 
• Carglumic acid

• For the treatment of pediatric and adult patients as adjunctive therapy to standard of care for acute hyperammonemia due to Nacetylglutamate synthase (NAGS) deficiency
• For the treatment of pediatric and adult patients as maintenance therapy for chronic hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency
• For the treatment of pediatric and adult patients as adjunctive therapy to standard of care for acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA).

Initial Criteria:

1. The patient has been diagnosed with hyperammonemia due to ONE of the following:

a. N-acetylglutamate synthase (NAGS) deficiency confirmed by either enzyme analysis (via liver biopsy) or genetic testing (medical record documentation required); OR

b. Propionic acidemia (PA) or Methylmalonic acidemia (MMA) (medical record documentation required); AND

2. The patient meets ALL of the following: 

a. Plasma ammonia level of 150 µmol/L (>260 µg/dl) or higher if a neonate (less than 28 days old) or > 100 µmol/L (>175 µg/dl) if an older child or adult (medical record documentation required); AND

b. The patient has a normal anion gap; AND 

c. The patient has a normal blood glucose level; AND

3. The patient is unable to maintain a plasma ammonia level within normal range with the use of a protein restricted diet and, when clinically appropriate, essential amino acid supplementation; AND

4. The prescribed dose and quantity are appropriate based on intended use and FDA labeling; AND 

5. If the request is for brand Carbaglu:

a. The patient had a sub-therapeutic or intolerant response (therapeutic failure) to an inactive ingredient of the generic product, but not present in the Brand (medical record documentation required); OR 

b. The patient has a documented intolerance to an inactive ingredient of the generic product that are not found in the brand product; (medical record documentation required); AND

6. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis; AND 

7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 365 days (1 year) 

1. The patient has been previously approved with Blue Cross and Blue Shield of North Carolina (Blue Cross NC) or would have met initial criteria for approval upon the start of therapy; AND

2. The patient has been able to achieve a plasma ammonia level within the normal range (medical record documentation required); AND

3. The prescribed dose and quantity are appropriate based on intended use and FDA labeling; AND

4. The prescriber is a specialist in the area of the patient’s diagnosis or the prescriber has consulted with a specialist in the area of the patient’s diagnosis; AND

5. If the request is for brand Carbaglu:

a. The patient had a sub-therapeutic or intolerant response (therapeutic failure) to an inactive ingredient of the generic product, but not present in the Brand; OR 

b. The patient has a documented intolerance to an inactive ingredient of the generic product that are not found in the brand product

Duration of Approval:  365 days (1 year)

all information referenced is from FDA package insert unless otherwise noted below. 

April 2022: Original utilization management policy issued.