FABHALTA (iptacopan)

FABHALTA (iptacopan)

Self-Administration – oral capsules

Diagnosis considered for coverage:

Paroxysmal Nocturnal Hemoglobinuria (PNH): Indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)

Coverage Criteria:

For diagnosis of PNH:

• Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH); AND
• Hemoglobin level of less than 10 g/dL; AND
• Prescribed by or in consultation with a hematologist or oncologist; AND
• Trial and inadequate response, contraindication, or intolerance to ONE of the following: 
  • Soliris 
  • Ultomiris 
  • Empaveli

Reauthorization Criteria:

For diagnosis of PNH:

• Patient demonstrates positive clinical response to therapy (e.g., hemoglobin stabilization, decrease in the number of red blood cell transfusions); AND
• Trial and inadequate response, contraindication, or intolerance to ONE of the following: 
  • Soliris
  • Ultomiris
  • Empaveli

Dosing:

• 200 mg orally twice daily 
• To reduce the potential risk of hemolysis with abrupt discontinuation of other PNH therapies:
  • For patients switching from Soliris (eculizumab), initiate FABHALTA no later than 1 week after the last dose of Soliris.
  • For patients switching from Ultomiris (ravulizumab), initiate FABHALTA no later than 6 weeks after the last dose of Ultomiris.
  • There is no available information regarding the timeframe for initiation of FABHALTA after other PNH therapies.

Coverage Duration: 

• Initial: 1 year
• Reauthorization: 1 year

Authorization is not covered for the following:

• The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• Fabhalta is the first FDA-approved oral treatment for adults with PNH.
• Fabhalta has a short half-life; nonadherent patients are at risk for serious breakthrough hemolysis (BTH) events with multiple missed doses.
• Fabhalta was studied in adults with PNH who had never been on a complement inhibitor before and in people who switched from C5 inhibitors.
• All agents in this class have a Boxed Warning for serious infections by encapsulated bacteria (N. meningitidis, S. pneumoniae, H. influenzae Type B). Complement inhibitors each have a REMS program that requires patients be vaccinated against pathogens of interest, unless the risks of delaying treatment outweigh the risks of infection.

Policy Updates:

• Effective 06/01/2024 – New program for Fabhalta approved by WHA P&T Committee (P&T, 05/21/24).

References:

1. Fabhalta Prescribing Information. Novartis Pharmaceuticals Corporation. East Hanover, New Jersey. December 2023.