Haemophilia B - KOL Insight

Extended half-life products have become the standard of care for haemophilia B patients, so what factors influence KOL prescribing decisions with respect to Sanofi/Sobi's Alprolix, CSL Behring's Idelvion and Novo Nordisk's Rebinyn/Refixia? While experts welcome the approval of CSL Behring/uniQure's gene therapy Hemgenix, will long-term safety concerns and cost limit its uptake? KOLs critically assess the prospects of launched and pipeline therapies.

Table of Contents

Executive summary (12)

Current and future treatment algorithm for haemophilia B

Research objectives (10)

Recombinant Factor IX (FIX) replacement therapies (28)

• Marketed therapies (28)
  • Standard half-life (SHL) products: BeneFIX (nonacog alfa; Pfizer), Ixinity (trenonacog alfa; Medexus Pharma) and Rixubis (nonacog gamma; Takeda) (8)
  • Extended half-life (EHL) products: Alprolix (eftrenonacog alfa; Sanofi/Sobi), Idelvion (albutrepenonacog alfa; CSL Behring) and Rebinyn/Refixia (nonacog beta pegol; Novo Nordisk) (13)
  • Bypassing agents: NovoSeven (eptacog alfa; Novo Nordisk), FEIBA (Factor Eight Inhibitor Bypassing Agent; Takeda), Sevenfact/Cevenfacta (coagulation Factor VIIa [recombinant]-jncw; LFB Biotechnologies/LFB USA) (7)

Gene therapy (21)

• Marketed therapies (13)
  • Hemgenix (etranacogene dezaparvovec; CSL Behring/uniQure) (13)
• Pipeline products (8)
  • Fidanacogene elaparvovec (Pfizer/Spark Therapeutics) (8)

Non-factor rebalancing agents (37)

• Pipeline products
• Anti-TFPI monoclonal antibodies (19)
  • Concizumab (Novo Nordisk) (13)
  • Marstacimab (Pfizer) (6)
• Antithrombin III inhibitors/RNA interference (9)
  • Fitusiran (Alnylam Pharmaceuticals/Sanofi) (9)
• Activated protein C receptor modulators (9)
  • SerpinPC (Centessa Pharmaceuticals) (9)

Future trends and opportunities in haemophilia B treatment (5)

• Key insights summary (5)

Appendix (4)

• KOL details (4)
  • KOLs from North America (1)
  • KOLs from Europe (2)