CurrentMass General Brigham Health PlanPolicy N/A

Pyrukynd (mitapivat) — coverage criteria for pyruvate kinase (PK) deficiency

This policy governs prior authorization, quantity/ specialty dispensing, and coverage criteria for Pyrukynd (mitapivat) for members with hemolytic anemia due to pyruvate kinase deficiency under Mass General Brigham Health Plan.

Policy Summary

PayerMass General Brigham Health Plan

PolicyPyrukynd (mitapivat) — coverage criteria for pyruvate kinase (PK) deficiency

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJun 1, 2023

Next Review DateN/A

Key ActionSubmit prior authorization and documentation confirming diagnosis of PK deficiency with genetic testing (≥2 PKLR mutant alleles with ≥1 missense) or enzyme assay, and evidence of clinical severity (Hb <10 g/dL or ≥6 transfusions in 12 months).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

7 monthsinitial approval duration

12 monthsreauthorization duration

6transfusion threshold

<10 g/dLhemoglobin threshold

≥2 allelesgenetic testing requirement

Coverage Criteria for Pyrukynd (mitapivat)

Initial therapy

Covered when ALL of the following are met

Main criteria: Physician documentation of diagnosis of hemolytic anemia with PK deficiency

Diagnostic confirmation (one required)

ONE of: Genetic testing demonstrating presence of at least 2 mutant alleles in the PKLR gene, of which at least 1 is a missense mutation
ONE of: Enzyme assay demonstrating deficiency in PK enzyme activity

Clinical severity (one required)

ONE of: Hemoglobin (Hb) level of < 10 g/dL< 10 g/dL
ONE of: Documentation of 6 blood transfusion episodes in the last 12 months>= 6 in last 12 months

Continuation of therapy

Reauthorization covered when

Reauthorization criteria: Diagnosis of hemolytic anemia with PK deficiency AND the member has achieved or maintained a positive clinical response to therapy (e.g., improvement in hemoglobin levels, reduction in blood transfusions)

Products obtained as samples or provided through a manufacturer's patient assistance program are excluded from authorization consideration and should not be counted toward prior authorization or continuity-of-care requests.

Key Clinical Thresholds and Coding

inv-04: Hemoglobin (Hb) — numeric threshold for alternative criterion

Hemoglobin (Hb) threshold< 10 g/dL

ContextUsed as an alternative clinical severity criterion for initial authorization of Pyrukynd (mitapivat)

Where foundCoverage criteria — hemoglobin requirement for treatment eligibility

inv-05: Transfusion episodes — numeric threshold (>= 6 in last 12 months)

Transfusion episodes threshold>= 6 blood transfusion episodes in the last 12 months

ContextUsed as an alternative clinical severity criterion for initial authorization of Pyrukynd (mitapivat)

Provider Requirements, Prior Authorization, and Dispensing

Prior Authorization

Prior Authorization Required

Prior authorization required. This product is designated specialty and must be filled at a contracted specialty pharmacy.

Prior authorization required for Pyrukynd (mitapivat).
Specialty dispensing: must be filled at a contracted specialty pharmacy.

Step Therapy

Step Therapy Note

Program type: Step therapy program is listed; no additional step requirements are detailed in this document.

Program type: Step Therapy (no specific prior steps documented in this policy).

Background on Pyrukynd (mitapivat)

Pyrukynd (mitapivat) is an FDA-approved therapy for adults with pyruvate kinase (PK) deficiency causing hemolytic anemia. Clinical benefit is demonstrated by improvement in hemoglobin levels and reduction in transfusion burden; prior authorization is required and continuity-of-therapy requests may be authorized for members already receiving Pyrukynd.

Definitions and Diagnostic Criteria

inv-12: Pyruvate kinase (PK) deficiency — hereditary cause of hemolytic anemia; diagnosis confirmed by genetic testing showing PKLR mutations.

DefinitionPyruvate kinase (PK) deficiency is a hereditary cause of hemolytic anemia for which Pyrukynd (mitapivat) is indicated; diagnosis can be confirmed by genetic testing showing PKLR mutations or by enzyme assay demonstrating deficient PK activity.

Genetic confirmation requirementGenetic testing demonstrating presence of at least 2 mutant alleles in the PKLR gene, with at least 1 missense mutation

Alternative diagnostic optionEnzyme assay demonstrating deficiency in PK enzyme activity

FDA indication notePyrukynd is indicated for the treatment of hemolytic anemia in adults with PK deficiency

Documentation Required

Required Clinical and Reauthorization Documentation

Physician documentation must confirm the diagnosis of hemolytic anemia due to pyruvate kinase (PK) deficiency and provide supporting genetic or enzyme testing results. For reauthorization, documentation must show the member has achieved or maintained a positive clinical response (for example, improved hemoglobin or reduced transfusion requirements).

Initial/ongoing documentation: Diagnosis of hemolytic anemia with PK deficiency and genetic testing showing ≥2 mutant PKLR alleles (at least one missense) or enzyme assay demonstrating PK deficiency.
Reauthorization documentation: Evidence of positive clinical response (e.g., improved hemoglobin levels or reduction in number of blood transfusions).