Piasky (crovalimab-akkz) — Medical Mutual

Initial criteria

• Patient is at least age ≥ 13 years; AND
• Patient does not have an unresolved serious Neisseria meningitidis infection prior to initiating therapy; AND
• Patient body weight is at least 40 kg; AND
• Patient must be vaccinated against meningococcal infection (serogroups A, C, W, Y, and B) according to current ACIP recommendations at least two weeks prior to initiation of therapy and will continue to be revaccinated in accordance with ACIP recommendations (OR if urgent therapy is indicated in a patient not up to date with vaccinations provide antibacterial prophylaxis and vaccinate as soon as possible); AND
• Will not be used in combination with another complement inhibitor therapy; AND
• For switch therapy: Patient is currently receiving treatment with eculizumab or ravulizumab and has shown a beneficial disease response and absence of unacceptable toxicity while on therapy; OR
• For treatment-naïve patient: Diagnosis confirmed by detection of PNH clones of at least 10% by flow cytometry diagnostic testing; AND
• Patient has the presence of at least 2 different glycosylphosphatidylinositol (GPI) protein deficiencies (e.g., CD55, CD59) within at least 2 different cell lines (e.g., granulocytes, monocytes, erythrocytes); AND
• Patient has laboratory evidence of significant intravascular hemolysis (LDH ≥ 1.5 x ULN) with symptomatic disease AND at least one of the following: symptomatic anemia (hemoglobin <7 g/dL OR <10 g/dL with cardiac symptoms on at least 2 measurements), thrombotic event related to PNH, organ damage secondary to chronic hemolysis (renal insufficiency, pulmonary insufficiency/hypertension), pregnant and potential benefit outweighs risk, disabling fatigue, abdominal pain requiring admission or opioid analgesia, dysphagia, or erectile dysfunction; AND
• Documented baseline values for one or more of the following: serum LDH, hemoglobin level, packed RBC transfusion requirement, history of thrombotic events

Reauthorization criteria

• Patient continues to meet universal and other indication-specific relevant criteria; AND
• Absence of unacceptable toxicity from the drug (examples: serious infections, severe infusion-related or injection-related reactions, anaphylaxis, etc.); AND
• Patient has not developed severe bone marrow failure syndrome (aplastic anemia or myelodysplastic syndrome) OR experienced a spontaneous disease remission OR received curative allogeneic stem cell transplant; AND
• Patient has shown a beneficial disease response compared to pretreatment baseline as indicated by one or more of: decrease in serum LDH, stabilization/increase in hemoglobin level, decrease in packed RBC transfusion requirement (≥30%), reduction in thromboembolic events

Approval duration

12 months