Next generation hemophilia therapies are aimed at revolutionizing the clinical outcomes and quality of life of hemophilia patients. Next generation therapies include bispecific antibodies, next generation recombinant products, and gene therapy. Bispecific antibodies mimic factor VIII or other molecules which function by inhibiting the natural anticoagulants such as antithrombin (AT) and tissue factor pathway inhibitor (TFPI), resulting in downstream hemostasis at the site of bleeding in patients with hemophilia. Next generation recombinant products are intended to extend the half-life of FVIII, resulting in longer infusion intervals. Gene therapy involves introducing genetic material into a patient’s body to deliver a correct copy of a gene to the cells to compensate for a defective one. The therapy is potentially transformational technology aimed at providing highly specialized, one-time treatment that addresses the root cause of the disease caused by genetic mutation.
Increase in initiatives for hemophilia awareness is a key factor driving the global next generation hemophilia therapies market. Hemophilia drug manufacturers and government and non-government organizations are focusing on nationwide campaigns to create awareness about the condition among the general population. Every year the World Federation of Hemophilia (WFH) conducts WFH Annual Global Survey, which aims to collect basic demographic information and data on access to care and treatment products in order to provide hemophilia treatment centers, health officials, and hemophilia organizations with useful and vital information to support efforts to improve or sustain the care to people with bleeding disorders. The aim of these programs and initiatives is to help people affected with hemophilia to seek proper support from health care professionals in addition to imparting awareness about the condition to the population at large in order to break down barriers and address stereotypes present in the society.
The current replacement therapy options used in the management of hemophilia have drawbacks such as frequent dosing with drugs requiring two to three times weekly infusions. This leads to patients developing neutralizing antibodies and also significantly adds to the cost of the treatment. In order to overcome these limitations, hemophilia drug manufacturers are entering into collaboration agreements with other players to develop novel treatment products such as bispecific antibodies, next generation recombinant factors, and gene therapies. In July 2018, Pfizer, Inc. launched phase III program with Spark Therapeutics to evaluate the effectiveness and safety of its FIX replacement gene therapy used in the treatment of hemophilia B.
The global next generation hemophilia therapies market can be segmented based on product type, disease indication, distribution channel, and region. In terms of product type, the next generation hemophilia therapies market can be divided into bispecific antibodies, next generation recombinant factors, and gene therapies. Based on disease indication, the global next generation hemophilia therapies market can be classified into hemophilia A, hemophilia B, and others. In terms of distribution channel, the next generation hemophilia therapies market can be divided into retail pharmacies, hospital pharmacies, and others.
In terms of region, the global next generation hemophilia therapies market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global next generation hemophilia therapies market in 2017 due to collaboration agreements by biopharmaceutical companies aimed at offering enhanced treatment options to manage hemophilia in patients. In May 2017, Pfizer, Inc. and Sangamo Therapeutics, Inc. entered into a global license and collaboration agreement to develop and commercialize gene therapy programs for hemophilia A which includes SB-525, the latter’s four lead product candidates. The aim of the collaboration agreement was to offer patients with highly specialized, one-time treatment options with gene therapy in order to address the root cause of the condition. Europe and Asia Pacific were the other major markets for next generation hemophilia therapies.
Prominent players operating in the global next generation hemophilia therapies market include Pfizer, Inc., Shire plc, Sanofi, F. Hoffmann-La Roche Ltd., and Novo Nordisk A/S.
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