Table of Contents
Increased use of recombinant and prophylaxis therapies drives growth
This research service focuses on the global haemophilia therapeutics market, with a particular focus on recently FDA-approved drugs and late-stage compounds for the treatment of haemophilia A, haemophilia B, inhibitors, and von Willebrand Disease. Pipeline analysis outlines the major FDA-approved haemophilia products, which include those submitted for regulatory review and those in Phase I, II, and III of clinical development. The study also provides an overview of the major clinical trials of recently approved or late-stage pipeline candidates. Novel technologies to extend product half-life and potential future therapies for haemophilia patients have also been discussed.
Executive Summary—Key Findings
- 2014 turned out to be a flagship year in the development of recombinant factor replacement therapies for haemophilia.
o Biogen Idec got Food and Drug Administration (FDA) approval for two of its recombinant therapies, FVIII (Eloctate) and FIX (Alprolix), in 2014.
o Baxter’s Obizur, which is a novel recombinant porcine FVIII product used to treat patients with acquired haemophilia A (a rare bleeding disorder) and inhibitors, gained FDA approval in October 2014.
o Biological License Application (BLA) was submitted for four recombinant FVII therapies, including BAX 111 (Baxter), BAX 855 (Baxter), BAY 81-8973 (Bayer), and Nuwiq® (Octapharma), as well as one recombinant FIX therapy, namely rIX-FP (CSL Behring).
- Within the next two years (2015 and 2016), in the United States alone nearly seven new products are expected to enter the haemophilia recombinant therapeutics market.
o The new products are likely to offer significant efficacy and moderate convenience benefits to patients, providing considerable incentives to switch to newer therapies, especially in the rFIX segment.
- The global haemophilia therapeutics market was valued at $ billion in 2014 and is projected to grow at a CAGR of % to reach $ billion in 2019.
o Increased uptake of recombinant therapies and prophylactic use, coupled with improved diagnosis of the disease, is expected to drive the growth of the global haemophilia therapeutics market.
o Recombinant therapies are likely to command a premium price and drive growth in the developed markets, while increased access to treatment and volumes per patient are likely to contribute to growth in the emerging markets.
o However, with several new recombinant products in the market, an increase in price pressure is anticipated, and negotiated discounts in emerging markets may drive down growth.
- Newer recombinant therapies are likely to gradually capture the share of the current leading participants, posing more of a long-term threat than a short-term one.
o Compelling convenience benefits are required for patients to switch therapies. None of the new rFVIII therapies has shown to extend half-life by more than %.
o The new market entrants such as Biogen Idec do not have well-established marketing/sales teams or good relationships with haematologists unlike the global big pharma companies.
o Moreover, global participants such as Bayer, Baxter, and Novo Nordisk have their own franchise of modified recombinant therapies in the pipeline to defend their market position.
- While the pipeline of recombinant replacement factors are maturing, other newer approaches are in the early phase of clinical development.
o Early proof-of-concept studies have shown that gene therapy can be a cost-effective alternative to treat haemophilia B patients.
o Companies such as Chatham Therapeutics and Amsterdam Molecular Therapeutics/uniQure are currently investigating new compounds in Phase I trials.
o Other novel therapeutics in early-stage pipeline include concizumab, a monoclonal antibody from Novo Nordisk, and ALN-AT3, an RNA interference therapeutic from Alnylam Pharmaceuticals.
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