Recombinant coagulation factors: market, products, R&D pipeline and new competition from alternative procoagulants and gene & cell therapy

STUTTGART, Germany I December 29, 2014 I 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 constantly grown to a size of US$ 7.4 bln in the year 2013. An oligopoly of five biopharmcaeutical companies essentially shared this sizeable market protected from competition by intellectual property. With the basic patents expiring now, an avenue for innovation has opened. Established key players as well as new stakeholder coming from technology providers or plasma-product companies have taken advantage and developed in a first wave new product candidates with improved properties regarding purity of the recombinant coagulation factor, longer half-life allowing less frequent injections and improved safety regarding immunogenicity.

Within the next two years, the most attractive market of recombinant coagulation factor VIII (rFVIII) with a market size of nearly US$ 5 bln in 2013, could see a total of seven new products if all of them were approved as planned. As a consequence, the rFVIII market will face a reshuffling of market players and market shares with newcomers as winners and established companies at risk to loose if improved rFVIII products or other recombinant coagulation factors cannot compensate for sales loss. The smaller recombinant coagulation factor IX market (US$ 832 mln with one established product) will be shared among one established and five new products with an expected rapid conversion from wild-type rFIX products to novel long-acting versions.

While R&D efforts to discover and develop novel next generation recombinant coagulation factors are successful in that new and improved products are coming to the market, there is still space for improvement. Especially truly long-acting rFVIII molecules have not yet been found, representing a great opportunity for innovators to generate clearly differentiated products.

While the R&D pipeline of classical recombinant coagulation factors is maturing, other approaches are strongly emerging in early clinical development which may become the real thread to the market of classical recombinant coagulation factors. Gene and cell therapy of hemophilia B and A has strongly gained in relevance by improved vectors and transgenes with a potentia total of 14 projects in early clinical trials by the end of 2015. However, gene therapy of hemophilia with the promise of cure by a single treatment course is a long-term plan from which not all patients will benefit due to preformed inhibitors. This problem might be overcome by the second emergent class of potential new treatments of hemophilia. Alternative procoagulants are in early clinicals studies which work as coagualation factor mimetics, as downstream coagulation factors or as a switch-off of inhibtors with a re-balance of the coagulation system in favor of a procoagulant state. Among the target profile of such alternative procoagualants are the possibility of subcutaneous administration, an universal use for treatment of hemophilia patients without and with inhibitors as well as reduced dosing frequency. The more of these properties are achived, the more likely such products could become blockbuster candidates.

Furthermore, immune tolerance inducing treatment modalities for hemophilia patients with inhibitors or as a prophylaxis to avoid inhibitor formation are progressing towards clinical evaluation.

With these new and emergent approaches also new stakeholders have entered the scenario of serious bleeding disorders. Among them are not only technology companies from the gene and cell therapy and RNAi field, but also from Big Pharma and rare disease specialist companies giving these new developments sufficient funding and power for determined development.

The report „Recombinant Coagulation Factors 2015: Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants“ from La Merie Publishing provides an update of recombinant coagulation factors. New sales data are analyzed to evaluate commercial development of the market under the light of a strong pipeline and entry of new competitor products into the market. The profiles of drug candidates in development are presented in detail. The competitve landscape is analyzed. A short- to mid-term outlook into the field is provided and the trends driving the future of this therapeutic segment are identified and described. The report was prepared by an experienced author with an independent view writing the fifth edition of this report series.

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SOURCE: La Merie Publishing