Pyrukynd – NC Standard

Restricted Products:

• Pyrukynd® (mitapivat)

FDA Approved Use:

• For the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

Criteria for Approval of Restricted Product(s):

Initial coverage:

1. The patient is 18 years of age or older; AND
2. The patient has been diagnosed with hemolytic anemia (medical record documentation required); AND
   1. The patient has confirmed pyruvate kinase deficiency (PKD) resulting from at least two variant alleles in the pyruvate kinase liver and red blood cell (PKLR) gene, of which at least one was a missense variant (medical record documentation required); AND
   2. The patient is NOT homozygous for the R479H mutation (medical record documentation required); AND
   3. The patient does NOT have two non-missense variants (without the presence of another missense variant) (medical record documentation required); AND
3. The patient has one of the following:
   1. The patient is regularly receiving RBC transfusions for hemolytic anemia due to PKD and has required a minimum of six within the previous year (medical record documentation required); OR
   2. The patient’s hemoglobin (Hb) level (within the last three months) is ≤10 g/dL (medical record documentation required); AND
4. The patient does not have moderate or severe hepatic impairment; AND
5. The patient will not be using in combination with any hematopoietic-stimulating agents; AND
6. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., hematologist); AND
7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 180 days

Continuation coverage:

1. The patient has been previously approved for Pyrukynd 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 continues to be managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., hematologist); AND
3. The therapy has been effective for the patient shown through ALL of the following:
   1. Patient reported improvements in fatigue and jaundice; AND
   2. Decrease in the need for blood transfusions; OR
      1. Hemoglobin response defined as ≥ 1.5 g/dL increase in hemoglobin concentration from baseline sustained at 2 or more scheduled assessments (medical record documentation required).

Duration of Approval: 365 days (1 year)

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: 365 days (1 year)

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 2025: Criteria change: Updated to allow approval for patients with the need of multiple RBC transfusions or hemoglobin (Hb) level is ≤10 g/dL. Added P&T statement.

March 2022: Original utilization management criteria issued.