Pyrukynd (mitapivat) — United Healthcare

Initial criteria

• Diagnosis of pyruvate kinase (PK) deficiency based on all of the following: presence of at least 2 variant alleles in the pyruvate kinase liver and red blood cell (PKLR) gene, of which at least 1 is a missense variant AND patient is not homozygous for the c.1436G>A (p.R479H) variant AND patient does not have 2 non-missense variants (without the presence of another missense variant) in the PKLR gene
• Used for the treatment of hemolytic anemia
• One of the following: (1) Both of the following: baseline hemoglobin ≤ 10 g/dL AND patient has had no more than 4 transfusions in the previous 52 weeks and no transfusions in the preceding 3-month period OR (2) patient has had a minimum of 6 transfusion episodes in the preceding 52 weeks
• Prescribed by a nephrologist or hematologist

Reauthorization criteria

• Documentation of positive clinical response to Pyrukynd therapy AND prescribed by, or in consultation with, a nephrologist or hematologist OR documentation does not provide evidence of positive clinical response to Pyrukynd therapy; allow for dose titration with discontinuation of therapy

Approval duration

Initial: 12 months; Reauthorization: 12 months if positive response, 4 weeks if titration/discontinuation