주요 업계 리더로부터의 인사이트에서는 혈우병 B 시장에서 유전자 치료제간의 경쟁 역학이 부각되고 있으며, Pfizer Beqvez/Durveqtix는 부신피질 스테로이드의 사용과 항체 문제에 의해 Hemgenix에 대한 과제에 직면하고 있습니다. Sanofi/Sobi의 Alprolix는 조기 시장 참여와 우수한 혈관 외 분포에 의해 반감기 연장 제품으로서 선호되고 있습니다. 또한 헴제닉스는 유망하기는 하나, 미국에서는 의료기관이 재정적 리스크에 신중한 것, 유럽에서는 가격 교섭으로 인해 도입이 늦는 것 등으로 인한 재정적·물류적 장벽이 채택을 방해하고 있습니다.
세계의 혈우병 B 시장에 대해 조사했으며, 시장의 개요와 출시 치료법, 파이프라인 동향, 향후 전망 등을 제공하고 있습니다.
목차
개요
현재 및 향후 치료 알고리즘
조사 목적
재조합 인자 IX(FIX) 보충 요법
승인된 약제
표준 반감기(SHL) 제품 : BeneFIX(nonacog alfa; Pfizer); Ixinity(trenonacog alfa; Medexus Pharma) and Rixubis(nonacog gamma; Takeda)
반감기 연장(EHL) 제품 : Alprolix(eftrenonacog alfa; Sanofi/Sobi); Idelvion(albutrepenonacog alfa; CSL Behring) and Rebinyn/Refixia(nonacog beta pegol; Novo Nordisk)
바이패스제 : NovoSeven(eptacog alfa; Novo Nordisk); FEIBA(Factor Eight Inhibiting Bypassing Agent; Takeda) and Sevenfact/Cevenfacta(coagulation Factor VIIa [recombinant]-jncw; LFB Biotechnologies/HEMA Biologics)
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
Table of Contents
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)
