Promacta – NC Standard

Restricted Products:

• Promacta® (eltrombopag) tablet
• Promacta® (eltrombopag) packet for suspension

FDA Approved Use:

• For the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• In combination with standard immunosuppressive therapy for the first-line treatment of adult and pediatric 2 years and older with severe aplastic anemia.
• For the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.
• For the treatment of thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy.

Criteria for INITIAL Approval of Restricted Product(s):

1. The patient has a diagnosis of chronic idiopathic thrombocytopenia (ITP); AND
   1. The patient has/had a platelet count ≤30 x 109/L at the start of therapy; AND
      1. The patient has a history of trial and failure of; or a documented intolerance, FDA labeled contraindication, or hypersensitivity to corticosteroids or immunoglobulins (IVIg or anti-D); OR
      2. The patient has had an insufficient response to or is not a candidate for splenectomy; OR
2. The patient has a diagnosis of severe aplastic anemia; AND
   1. At least two of the following:
      1. Neutrophils less than 0.5 X 109/L; OR
      2. Platelets less than 20 X 109/L; OR
      3. Reticulocytes less than 1% corrected (percentage of actual hematocrit [Hct] to normal Hct) or reticulocyte count <20 X 109/L; AND
   2. At least one of the following:
      1. Severe hypocellularity: <25%; OR
      2. Moderate hypocellularity, 25-50% with hematopoietic cells representing less than 30% of residual cells; AND
   3. At least one of the following:
      1. The patient will use the requested agent as first-line treatment AND the patient will be using the requested agent in the combination with standard immunosuppressive therapy (i.e.; antithymocyte globulin (ATG) AND cyclosporine); OR
      2. The patient has tried and failed or has a clinical intolerance/contraindication to standard immunosuppressive therapy (i.e.; antithymocyte globulin (ATG) AND cyclosporine); OR
3. The patient has a diagnosis of hepatitis C associated thrombocytopenia; AND
   1. The patient’s platelet count is <75 x109/L AND the intent is to increase platelet counts sufficiently to initiate interferon therapy; OR
   2. The patient is on concurrent therapy with interferon AND is at risk for discontinuing HCV therapy due to thrombocytopenia; AND
4. If the requested medication is Promacta packet for suspension; AND
   1. The provider attests that the patient is unable to take solid dosage forms; AND
   2. The patient is not taking any other medication in a solid dosage form; AND
5. The patient will not be using Promacta in combination with another Thrombopoietin Receptor Agonists (e.g., Alvaiz, Mulpleta, Nplate, Doptelet, Tavalisse); AND
6. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval:

chronic idiopathic thrombocytopenia (ITP) 8 weeks

severe aplastic anemia 16 weeks

hepatitis C associated thrombocytopenia 12 weeks

Criteria for CONTINUED Approval of Restricted Product(s):

1. The patient has a diagnosis of chronic idiopathic thrombocytopenia (ITP); AND
   1. The patient was approved for coverage through the initial coverage criteria above; AND
      1. The patient’s platelet count is ≥50 x 109/L; OR
      2. The patient’s platelet count has increased sufficiently to avoid clinically important bleeding; OR
2. The patient has a diagnosis of severe aplastic anemia; AND
   1. The patient was approved for coverage through the initial coverage criteria above; AND
   2. The patient has had a hematological response by week 16 defined as ONE of the following:
      1. Platelet count increases to 20 x 109/L above baseline: OR
      2. Stable platelet counts with transfusion independence for a minimum of 8 weeks; OR
      3. Hemoglobin increase by greater than 1.5 g/dL; OR
      4. Reduction in greater than or equal to 4 units of Red Blood Cell (RBC) transfusions for 8 consecutive weeks; OR
      5. An Absolute Neutrophil Count (ANC) increase of 100%; OR
      6. An Absolute Neutrophil Count (ANC) increase greater than 0.5 x 109/L. OR
3. The patient has a diagnosis of hepatitis C associated thrombocytopenia; AND
   1. The patient was approved for coverage through the initial coverage criteria above; AND
   2. The patient will be initiating hepatitis C therapy with interferon; OR
   3. The patient will be maintaining hepatitis C therapy with interferon at the same time as Promacta (eltrombopag); AND
4. If the requested medication is Promacta packet for suspension; AND
   1. The provider attests that the patient is unable to take solid dosage forms; AND
   2. The patient is not taking any other medication in a solid dosage form.
5. The patient will not be using Promacta in combination with another Thrombopoietin Receptor Agonists (e.g., Alvaiz, Mulpleta, Nplate, Doptelet, Tavalisse)

Duration of Approval:

chronic idiopathic thrombocytopenia (ITP) 365 days

severe aplastic anemia 365 days

hepatitis C associated thrombocytopenia 48 weeks

Quantity Limitations:

Quantity limitations apply to brand and associated generic products.

Quantity Limit Exception Criteria:

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed).

Duration of approval:

chronic idiopathic thrombocytopenia (ITP) 365 days

severe aplastic anemia 365 days

hepatitis C associated thrombocytopenia 48 weeks

References:

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

Policy Implementation/Update Information:

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q2 annually.

February 2024: Criteria update: Added Alvaiz to list of Thrombopoietin Receptor Agonist that cannot be used in combination with Promacta.

April 2022: Criteria change: Added Promacta cannot be used in combination with another Thrombopoietin Receptor Agonist. Added that patient will be required to have tried and failed or has a clinical intolerance/contraindication to standard immunosuppressive therapy (i.e.; antithymocyte globulin (ATG) AND cyclosporine) for severe aplastic anemia;

January 2022: Criteria change: Annual criteria review. Addition of quantity limit.

January 2019: Added new formulation Promacta powder for suspension to the criteria

November 2018: Reformat criteria and added new indication for 1st line treatment of severe aplastic anemia in adults and pediatrics 2 years & older

July 2016: Original utilization management criteria issued.