Empaveli- Fabhalta- Voydeya – NC Standard

Restricted Product(s)

• Empaveli® (pegcetacoplan) 
• Fabhalta® (iptacopan) 
• VoydeyaTM (danicopan)

FDA Approved Use for Empaveli

• For the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)

FDA Approved Use for Fabhalta

• For the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)
• For the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g
  • This indication is approved under accelerated approval based on reduction of proteinuria. It has not been established whether Fabhalta slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.
• For the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria.

FDA Approved Use for Voydeya

• For add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) 
• Limitations of Use: Voydeya has not been shown to be effective as monotherapy and should only be prescribed as an add-on to ravulizumab or eculizumab

Criteria for Approval of Restricted Product(s)

Initial Criteria for Approval

1. The patient is 18 years of age or older; AND 
2. The patient has a confirmed diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) (medical record documentation required); AND
   1. The request is for Empaveli; AND 
      1. ONE of the following: 
         1. The patient has a hemoglobin ≤ 7 g/dL (medical record documentation required); OR 
         2. The patient has hemoglobin ≤ 9 g/dL and is experiencing symptoms of anemia (medical record documentation required); OR 
         3. The patient has documented symptoms of thromboembolic complications (abdominal pain, shortness of breath, chest pain, end organ damage) (medical record documentation required); OR 
         4. The patient is transitioning from another complement inhibitor approved for the diagnosis of PNH (e.g. eculizumab or ravulizumab); AND
            1. The patient was previously approved through Blue Cross NC initial criteria for eculizumab or ravulizumab; AND 
      2. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g., crovalimab, eculizumab, ravulizumab, iptacopan); OR 
         1. The patient is transitioning from eculizumab to pegcetacoplan; AND 
         2. The patient will discontinue eculizumab 4 weeks after initiation of pegcetacoplan therapy; OR 
   2. The request is for Fabhalta; AND 
      1. ONE of the following:
         1. The patient has a hemoglobin < 10 g/dL (medical record documentation required); AND 
            1. The patient has a lactate dehydrogenase (LDH) > 1.5 times upper limit of normal (medical record documentation required); OR 
         2. The patient is transitioning from another complement inhibitor approved for the diagnosis of PNH (e.g. eculizumab or ravulizumab); AND 
            1. The patient was previously approved through Blue Cross NC initial criteria for eculizumab or ravulizumab; AND 
      2. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g., crovalimab, eculizumab, ravulizumab, pegcetacoplan); OR 
         1. The patient is transitioning from eculizumab to iptacopan; AND 
            1. The patient will initiate iptacopan no later than 1 week after the last dose of eculizumab; OR 
         2. The patient is transitioning from ravulizumab to iptacopan; AND 
            1. The patient will initiate iptacopan no later than 6 weeks after the last dose of ravulizumab; OR 
   3. The request is for Voydeya; AND 
      1. The patient has hemoglobin less than 10 g/dL and is experiencing symptoms of anemia (medical record documentation required); AND 
      2. The patient is being treated for extravascular hemolysis; AND 
      3. The requested medication will be used as add on therapy with eculizumab or ravulizumab; OR
3. The patient has a confirmed diagnosis of primary immunoglobulin A nephropathy (IgAN) (medical record documentation required); AND
   1. The request is for Fabhalta; AND 
   2. The patient has a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g (medical record documentation required); OR
4. The patient has a confirmed diagnosis of complement 3 glomerulopathy (C3G) (medical record documentation required); AND
   1. The request is for Fabhalta; AND
   2. The patient has a urine protein-to-creatinine ratio (UPCR) ≥1 g/g (medical record documentation required); AND
   3. The patient has patient has tried and failed or has a clinical intolerance/contraindication to angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days (medical record documentation required); AND
   4. The patient has a reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) (medical record documentation required); AND
   5. The patient has a measured GFR ≥ 30 ml/min/1.73m2 (medical record documentation required); AND
5. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hematologist, oncologist, or immunology specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis; AND
6. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 180 days (6 months)

Continuation Criteria for Approval

1. The patient has been previously approved with Blue Cross and Blue Shield of North Carolina (Blue Cross NC) or would have met initial criteria for approval upon the start of therapy (medical record documentation required); AND 
2. The patient has experienced stabilization or improvement of symptoms from baseline while using the requested agent, as demonstrated by the following (medical record documentation required):
   1. Patient has diagnosis of paroxysmal nocturnal hemoglobinuria (PNH): AND 
      1. Significant reduction in transfusion requirements (medical record documentation required); AND 
      2. No thromboembolism events persisting while using the requested agent (medical record documentation required); OR 
   2. Patient has a diagnosis of primary immunoglobulin A nephropathy (IgAN) OR complement 3 glomerulopathy (C3G); AND 
      1. Improvement in patient’s urine protein-to-creatinine ratio (UPCR) (medical record documentation required); AND 
3. The request is for Empaveli or Fabhalta; AND 
   1. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g. crovalimab, eculizumab, ravulizumab, iptacopan, pegcetacoplan) (medical record documentation required).

Duration of Approval: 365 days (1 year)

Quantity Limit Exception Criteria

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND 
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR 
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed).
   
   Duration of Approval: 365 days (1 year)

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q2 annually.

April 2025: Criteria change (Fabhalta): Added new indication for the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria.

September 2024: Criteria update: Added crovalimab to list of complement inhibitors not to be used concomitantly with requested medication and added limitations of use to FDA section.

August 2024: Criteria change (Fabhalta): Added new expanded indication for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g.

June 2024: Criteria update (Voydeya): Updated required hemoglobin level for approval of Voydeya.

April 2024: Criteria change: Added new to market Voydeya to policy. Changed name of policy to include Voydeya in title. Quantity limit section updated.

December 2023: Criteria change: Added new to market Fabhalta to policy. Changed name of policy to include Fabhalta in title. Added P&T Review statement to policy.

June 2023: Criteria update: Criteria review and formatting changes. Added duration of approval to quantity limit exception criteria.

June 2021: Original utilization management policy issued.