Ultomiris™

Drug - Ultomiris™ (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

Questions?

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