Empaveli (pegcetacoplan) — Medical Mutual

Initial criteria

• Patient age ≥ 18 years
• Patient does not have an unresolved, serious systemic infection from encapsulated bacteria prior to initiating therapy
• Patient must be vaccinated against encapsulated bacteria (e.g., Streptococcus pneumoniae, Neisseria meningitidis [serogroups A, C, W, Y and B], Haemophilus influenzae type b, etc.) according to current ACIP recommendations at least two weeks prior to initiation and will continue revaccination per ACIP OR, if urgent therapy is indicated, antibacterial prophylaxis and vaccines administered as soon as possible
• Will not be used in combination with another complement inhibitor therapy (except a 4-week run-in period allowed when transitioning from eculizumab to pegcetacoplan)
• Used as switch therapy AND patient is currently receiving treatment with eculizumab or ravulizumab and has shown beneficial disease response and absence of unacceptable toxicity while on therapy; OR
• Patient is complement inhibitor treatment-naïve AND diagnosis confirmed by detection of PNH clones of at least 10% by flow cytometry diagnostic testing AND patient has at least 2 different glycosylphosphatidylinositol (GPI) protein deficiencies within at least 2 different cell lines 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 hemoglobin < 10 g/dL in at least two independent measurements in a patient with cardiac symptoms)
• - Presence of a thrombotic event related to PNH
• - Presence of organ damage secondary to chronic hemolysis (renal insufficiency, pulmonary insufficiency/hypertension)
• - Patient is pregnant and potential benefit outweighs potential fetal risk
• - Patient has disabling fatigue
• - Abdominal pain requiring admission or opioid analgesia, dysphagia, or erectile dysfunction
• Documented baseline values required: serum lactate dehydrogenase (LDH), hemoglobin level, packed RBC transfusion requirement, history of thrombotic events

Reauthorization criteria

• Patient continues to meet universal and indication-specific criteria
• Absence of unacceptable toxicity from the drug (e.g., serious infections from encapsulated bacteria, severe infusion-related/hypersensitivity reactions)
• Patient has not developed severe bone marrow failure syndrome (aplastic anemia or myelodysplastic syndrome) OR experienced spontaneous disease remission OR received curative allogeneic stem cell transplant
• Patient has shown a beneficial disease response compared to pretreatment baseline indicated by one or more of: decrease in serum LDH, stabilization/increase in hemoglobin level, decrease in packed RBC transfusion requirement (reduction of at least 30%), reduction in thromboembolic events
• Dose escalation (up to maximum allowed) may occur if patient has shown an initial response, received initial dose and interval for at least 4 weeks, AND LDH levels remain >2x ULN despite treatment

Approval duration

12 months