Fabhalta (iptacopan) — Highmark

Initial criteria

• age ≥ 18 years
• diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) (ICD-10: D59.5)
• PNH mutant clones confirmed by flow cytometry OR glycosylphosphatidylinositol-anchored protein (GPI-AP)-deficient polymorphonuclear cells (PMNs) confirmed by flow cytometry
• baseline hemoglobin level < 10.5 g/dL
• elevated lactate dehydrogenase (LDH) ≥ 1.5 × ULN OR history of a thromboembolic event (e.g., DVT, PE) OR clinical findings of systemic complications (e.g., fatigue, hemoglobinuria, abdominal pain, dyspnea, dysphagia, erectile dysfunction, history of blood cell transfusion due to PNH)
• Empaveli will not be used in combination with another complement inhibitor for PNH (e.g., Soliris, Ultomiris, Fabhalta), unless cross-titrating from Soliris or Ultomiris to Empaveli; Fabhalta will not be used in combination with another complement inhibitor for PNH (e.g., Soliris, Ultomiris, Empaveli), unless cross-titrating