Hemophilia Pipeline Overview – Once-a-week prophylaxis may soon become a reality

• Publication date: November 2012
• Report size: 30 pages

Among the products for hemophilia A and B that are likely to reach the market within the next 4 years are modified recombinant factor replacements that could reduce injection frequencies significantly, making once-a-week prophylaxis a reality.Assess key pipeline products with regards to current gold-standard therapies.Identify the prosp...

Among the products for hemophilia A and B that are likely to reach the market within the next 4 years are modified recombinant factor replacements that could reduce injection frequencies significantly, making once-a-week prophylaxis a reality.Assess key pipeline products with regards to current gold-standard therapies.Identify the prospects faced by next-generation products in this niche market.Review and profile current key pipeline hemophilia A and B factor replacement products.Assess current unmet needs and challenges faced by pipeline products.The most important unmet need is a longer half-life for clotting factors so that daily intravenous injections are no longer necessary. Moving from daily to weekly or fortnightly injections would improve patients’ quality of life enormously.Since longer clotting factor half-life is the major unmet need in hemophilia treatment, Datamonitor believes that novel recombinant compounds can only gain significant market share by either addressing the lack of long-acting recombinant factor IX therapeutics or by addressing the need for more competitively priced recombinant products.Economy of scale plays a role when producing a biologic compound cost-effectively and established companies will have an advantage in this area; having the right infrastructure, production facilities, marketing channels, and sales force are important factors that will determine whether a company can successfully enter the hemophilia market.Datamonitor’s up to date key pipeline hemophilia A and B therapeutics profiles includes the latest clinical data and industry developments.Understand the most pressing unmet needs in hemophilia and how current pipeline products can gain significant market share by addressing them.Compare pipeline products with current gold-standard treatments and assess what they will have to do to deliver growth.

Pathology Industry

OVERVIEW

Catalyst
Summary
Clinical Pipeline Overview
A handful of key players dominate the market
Gene therapy looks hopeful but market entry is still not predicted for this decade
Current Gold-Standard Product Profiles
Kogenate FS (FVIII recombinant; Bayer): the gold-standard hemophilia A therapy
BeneFIX (FIX recombinant; Pfizer): the gold-standard hemophilia B therapy
Target product profile versus current level of attainment
Key Factor VIII Pipeline Products
NN7008 (turoctocog alfa; Novo Nordisk)
BIIB 031 (rFVIIIFc; Biogen Idec)
NN7088 (rFVIII, glycopegylated; Novo Nordisk)
BAY-94-9027 (recombinant PEGylated factor VIII; Bayer)
Clinical and commercial attractiveness for key hemophilia A pipeline products
Key Factor IX Pipeline Products
BAX 326 (recombinant PEGylated factor IX; Baxter)
CSL654 (rFIX-albumin fusion; CSL Behring)
BIIB 029 (rFIXFc; Biogen Idec)
NN7999 (rFIX, glycoPEGylated; Novo Nordisk)
Clinical and commercial attractiveness for key hemophilia B pipeline products
Bibliography
Journal articles and online sources
Appendix
Methodology
Datamonitor drug assessment for clinical and commercial attractiveness


TABLES

Table: Clinical pipeline key drugs for hemophilia A and B, November 2012
Table: Kogenate FS (recombinant Factor VIII; Bayer) – drug profile, 2012
Table: Kogenate FS (recombinant Factor VIII; Bayer) – drug profile, 2012
Table: Kogenate FS (rFVIII; Bayer) and BeneFIX (rFIX; Pfizer) vs the target product profile in previously treated patients, 2012
Table: Key events in the clinical development of NN7008 (turoctocog alfa; Novo Nordisk), 2012
Table: Key events in the clinical development of BIIB 031 (rFVIIIFc; Biogen Idec), 2012
Table: Key events for the clinical development of NN7088 (Novo Nordisk), 2012
Table: Key events for the clinical development of BAY-94-9027 (Bayer), 2012
Table: Key events for the clinical development of BAX 326 (Baxter), 2012
Table: Key events for the clinical development of CSL654 (CSL Behring), 2012
Table: Key events for the clinical development of BIIB 029 (rFIXFc; Biogen Idec), 2012
Table: Key events for the clinical development of NN7999 (Novo Nordisk), 2012


FIGURES

Figure: Clinical and commercial attractiveness for key hemophilia A pipeline drugs vs comparator Kogenate FS (rFVIII; Bayer), 2012
Figure: Breakdown of clinical and commercial attractiveness attributes for key hemophilia A pipeline drugs, 2012
Figure: Launch timeline for key hemophilia A pipeline drugs, 2012
Figure: Clinical and commercial attractiveness for key hemophilia B pipeline drugs vs comparator BeneFIX (rFIX; Pfizer), 2012
Figure: Breakdown of clinical and commercial attractiveness attributes for key hemophilia B pipeline drugs, 2012
Figure: Launch timeline for key hemophilia B pipeline drugs, 2012

Companies Mentioned
Arriva plc, CSL Behring, Hutchison 3G UK Limited, Imperial Tobacco Group PLC, Ipsen S.A., Nektar Therapeutics, Telstra Corporation Limited