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DrugUltomiris™ (ravulizumab solution, concentrate) [Alexion Pharmaceuticals Inc.]

July 2019

Therapeutic area - Monoclonal Antibody; Monoclonal Antibody, complement Inhibitor

Initial approval criteria

  • Patient must be ≥ 18 years old AND
  • Patient has a diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) AND
  • Diagnosis must be accompanied by detection of PNH clones of at least 5% by flow cytometry diagnostic testing AND demonstrate the presence of at least 2 different glycosylphosphatidylinositol (GPI) protein deficiencies (e.g., CD55, CD59, etc.) within at least 2 different cell lines (granulocytes, monocytes, erythrocytes) AND
  • Patient has at least ONE of the following indications for therapy:
    • Presence of a thrombotic event
    • Presence of organ damage secondary to chronic hemolysis
    • Patient is pregnant and potential benefit outweighs potential fetal risk
    • Patient is transfusion dependent
    • Patient has high LDH activity (defined as ≥1.5 x ULN) with clinical symptoms
  • Patient must have documented baseline values for 1 or more of the following (necessary for renewal): serum lactate dehydrogenase (LDH), hemoglobin level, and packed RBC transfusion requirement AND
  • Patient has tried/failed eculizumab, unless contraindicated AND
  • Will NOT be used in combination with eculizumab AND
  • Patient does not have a systemic infection AND
  • Patient does not have an unresolved Neisseria meningitidis infection AND
  • Vaccination or revaccination for meningococcal disease according to the most current Advisory Committee on Immunization Practices (ACIP) has been performed ≥ 2 weeks prior to initiation of therapy AND
  • If vaccines are administered < 2 weeks prior to ravulizumab therapy, 2 weeks of antibacterial drug prophylaxis should be prescribed AND
  • Prescriber is enrolled in the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) program

Renewal criteria

  • Prescriber is enrolled in the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) program AND
  • Patient meets the above criteria AND
  • Patient does not have unacceptable toxicity such as serious meningococcal infections (septicemia and/or meningitis), infusion reactions, serious infections, thrombotic microangiopathy complications (TMA), etc. AND
  • Patient demonstrates improvement or stabilization of PNH disease by one of the following: decrease in serum LDH from pretreatment baseline, stabilization/improvement in hemoglobin level from pretreatment baseline, decrease in packed RBC transfusion requirement from pretreatment baseline

Quantity limits

  • Loading dose: ≤ 10 vials (300 mg/30 mL single-dose vials) x 1 time
  • Maintenance dose: ≤ 12 vials every 8 weeks, starting 2 weeks after loading dose


MHCP Provider Call Center 651-431-2700 or 800-366-5411

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