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Hemophilia Market Likely to Undergo Major Changes

As key patents expire, researchers are exploring new and better treatments

Major changes lie in store for the market that focuses on the treatment of hemophilia, according to an analysis by La Merie Publishing, a biopharmaceutical consultant based in Germany.

Since approval of the first recombinant coagulation factors for treatment and prophylaxis of hemophilia A and B about two decades ago, the market of recombinant coagulation factors has grown constantly, reaching $7.4 billion in 2013. Five companies essentially shared this market, protected from competition by basic patents that are now expiring. New product candidates have improved purity, longer half-lives that allow less frequent injections, and improved safety regarding immunogenicity.

Within the next two years, the most attractive market — recombinant coagulation factor VIII (rFVIII), with a market size of nearly $5 billion in 2013 — could see seven new products. As a consequence, this market will face a reshuffling of market players and shares. The smaller recombinant coagulation factor IX market ($832 million with one established product) will be shared by five new products, including novel long-acting versions.

While the pipeline of classical recombinant coagulation factors is maturing, other approaches are in early clinical development. Gene and cell therapy for hemophilia B and A has gained relevance through improved vectors and transgenes; 14 projects may be in early clinical trials by the end of 2015. However, gene therapy for hemophilia with the promise of a cure by a single treatment course is a long-term prospect.

Another emerging class of potential new hemophilia treatments consists of alternative procoagulants (now in early clinical studies) that work as coagulation factor mimetics, as downstream coagulation factors, or as “off switches” for inhibitors. Such alternative procoagulants may offer subcutaneous administration and reduced dosing frequency.

La Merie’s report, “Recombinant Coagulation Factors 2015: Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants,” provides new sales data, drug candidate profiles, competitive landscape, and trends driving the future.

Source: La Merie Publishing; December 29, 2014. 

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