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Hemophilia Pipeline Overview - Once-a-week prophylaxis may soon become a reality
Date:12/13/2012

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.

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

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1. Novo Nordisk introduces HemaGo, a mobile application to track hemophilia treatment, bleeding episodes and life events
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3. Inspiration Biopharmaceuticals Announces Clinical Hold of Clinical Trials Evaluating IB1001 for the Treatment and Prevention of Bleeding in Hemophilia B
4. Inspiration Biopharmaceuticals Reports Positive Interim Results from the Accur8 Phase 2/3 Study of OBI-1 in Acquired Hemophilia A at WFH 2012 World Congress
5. CSL Behring Receives FDA Orphan Drug Designation for rIX-FP, a Novel Therapy in Development to Treat Congenital Factor IX Deficiency (Hemophilia B)
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