Prior Authorization and Quantity Limit Criteria for Approval

Initial Evaluation

Miglustat and Yargesa will be approved when ALL of the following are met:

1. ONE of the following:
   1. ALL of the following:
      1. The patient has a diagnosis of Gaucher disease type 1 (GD1) confirmed by ONE of the following:
         1. A baseline (prior to therapy for the requested indication) glucocerebrosidase enzyme activity of less than or equal to 15% of mean normal in peripheral blood leukocytes, fibroblasts, or other nucleated cells
            OR
         2. Confirmation of genetic mutation of the glucocerebrosidase (GBA) gene with two disease-causing alleles
            AND
      2. The prescriber has drawn baseline (prior to therapy for the requested indication) measurements of hemoglobin level, platelet count, liver volume, and spleen volume
         AND
      3. Prior to any treatment for the intended diagnosis, the patient has had at least ONE of the following clinical presentations:
         
         1. Anemia [defined as mean hemoglobin (Hb) level below the testing laboratory’s lower limit of the normal range based on age and gender]
            OR
         2. Thrombocytopenia (defined as platelet count of less than 100,000 per microliter)
            OR
         3. Hepatomegaly
            OR
         4. Splenomegaly
            OR
         5. Growth failure (i.e., growth velocity is below the standard mean for age)
            OR
         6. Evidence of bone disease with other causes ruled out
            OR
   2. ALL of the following:
      1. The patient has a diagnosis of Niemann-Pick disease type C (NPC) as confirmed by genetic analysis mutation in the NPC1 or NPC2 genes
         AND
      2. The requested medication will be used for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC)
         AND
      3. The requested medication will be used in combination with Miplyffa (arimoclomol)
         AND
2. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, gastroenterologist, geneticist, hematologist, hepatologist, neurologist, specialist in metabolic diseases) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
   AND
3. ONE of the following:
   1. The requested quantity (dose) does NOT exceed the program quantity limit
      OR
   2. BOTH of the following:
      1. The requested quantity (dose) is greater than the program quantity limit
         AND
      2. The prescriber has provided information in support of therapy with a higher dose for the requested indication

Length of Approval: 12 months

Renewal Evaluation

Miglustat and Yargesa will be approved when ALL of the following are met:

1. The patient has been previously approved for the requested medication through the plan’s Prior Authorization criteria
   AND
2. ONE of the following:
   1. BOTH of the following:
      1. The patient has a diagnosis of Gaucher disease type 1 (GD1)
         AND
      2. The patient has had improvements or stabilization with the requested medication as indicated by ONE of the following:
         1. Spleen voulme
            OR
         2. Hemoglobin level
            OR
         3. Liver volume
            OR
         4. Platelet count
            OR
         5. Growth
            OR
         6. Bone pain or crisis
            OR
   2. ALL of the following:
      1. The patient has a diagnosis of Niemann-Pick disease Type C (NPC)
         AND
      2. The requested medication will be used for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC)
         AND
      3. The requested medication will be used in combination with Miplyffa (arimoclomol)
         AND
      4. The patient has had clinical benefit with the requested medication
         AND
3. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, gastroenterologist, geneticist, hematologist, hepatologist, neurologist, specialist in metabolic diseases) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
   AND
4. ONE of the following:
   1. The requested quantity (dose) does NOT exceed the program quantity limit
      OR
   2. BOTH of the following:
      1. The requested quantity (dose) is greater than the program quantity limit
         AND
      2. The prescriber has provided information in support of therapy with a higher dose for the requested indication

Length of Approval: 12 months