Haemophilia B - KOL Insight

Description

This report provides an in-depth analysis of the haemophilia B treatment landscape, focusing on the evolving use of standard and extended half-life products. Insights from key opinion leaders highlight the competitive dynamics between gene therapies, with Pfizer's Beqvez/Durveqtix facing challenges against Hemgenix due to corticosteroid use and antibody issues. Sanofi/Sobi's Alprolix emerges as the preferred extended half-life product, favoured for its early market entry and superior extravascular distribution. Additionally, while Hemgenix shows promise, its adoption is hindered by financial and logistical barriers, with institutions cautious about the financial risks in the U.S. and pricing negotiations slowing its rollout in Europe.

Key questions answered:

How do experts use and prefer standard half-life (SHL) and extended half-life (EHL) products?
What changes are expected in the use of SHL and EHL products over the next 3-5 years?
What are the expert views on bypassing agents and their future role?
How is CSL Behring/uniQure's Hemgenix perceived post-approval?
What are the anticipated barriers to the uptake of gene therapies?
How are non-factor rebalancing therapies, like Alhemo and Hympavzi, viewed?
Are there safety concerns associated with non-factor therapies?
How will the haemophilia B treatment landscape evolve in the coming years?

Key brands covered in this report:

BeneFIX (nonacog alfa)
Ixinity (trenonacog alfa)
Rixubis (nonacog gamma)
Alprolix (eftrenonacog alfa)
Idelvion (albutrepenonacog alfa)
Rebinyn/Refixia (nonacog beta pegol)
NovoSeven (eptacog alfa)
FEIBA (Factor Eight Inhibiting Bypassing Agent)

Sevenfact/Cevenfacta (coagulation Factor VIIa [recombinant]-jncw)
Hemgenix (etranacogene dezaparvovec)
Beqvez/Durveqtix (fidanacogene elaparvovec)
Alhemo (concizumab)
Hympavzi (marstacimab)
fitusiran
SerpinPC

Companies:

Sanofi
Pfizer
Takeda
CSL Behring
Novo Nordisk
Alnylam Pharmaceuticals

Centessa Pharmaceuticals
HEMA Biologics
LFB Biotechnologies
Sobi
uniQure
Medexus

Executive summary

Current and future treatment algorithm

Research objectives

Recombinant Factor IX (FIX) replacement therapies

Approved drugs
- Standard half-life (SHL) products: BeneFIX (nonacog alfa; Pfizer); Ixinity (trenonacog alfa; Medexus Pharma) and Rixubis (nonacog gamma; Takeda)
- Extended half-life (EHL) products: Alprolix (eftrenonacog alfa; Sanofi/Sobi); Idelvion (albutrepenonacog alfa; CSL Behring) and Rebinyn/Refixia (nonacog beta pegol; Novo Nordisk)
- Bypassing agents: NovoSeven (eptacog alfa; Novo Nordisk); FEIBA (Factor Eight Inhibiting Bypassing Agent; Takeda) and Sevenfact/Cevenfacta (coagulation Factor VIIa [recombinant]-jncw; LFB Biotechnologies/HEMA Biologics)

Gene therapy

Approved drugs
- Hemgenix (etranacogene dezaparvovec; uniQure/CSL Behring)
- Beqvez/Durveqtix (fidanacogene elaparvovec; Pfizer)

Non-factor rebalancing agents

Approved drugs
- Hympavzi (marstacimab; Pfizer)
Pipeline drugs
- Alhemo (concizumab; Novo Nordisk)
- Fitusiran (Alnylam Pharmaceuticals/Sanofi)
- SerpinPC (Centessa Pharmaceuticals)