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Products in the pipeline

Never have so many coagulation therapies been in development or “in the pipeline”. So many, in fact, that it has become difficult to keep track of them all. So the CHS is publishing four charts, one each for clotting factor concentrates, inhibitor products, non-factor coagulation products that may prove efficacious in both inhibitor and non-inhibitor patients, and gene therapy, to help everyone stay informed of their progress through clinical trials and regulatory approval.

In addition to the large number of therapies recently introduced (see www.hemophilia.ca/en/bleeding-disorders/clotting-factor-concentrates), we have identified another 21 new therapies in development or soon to be marketed, including nine clotting factor concentrates, four bypassing therapies to treat patients with inhibitors, two non-factor coagulation products and six gene therapy products. While some of these are still in early Phase I or II stages in the research process and several years away entering the market, others are in, or have completed, Phase III trials with patients, the final stage before an application is made to market the product commercially.

Health Canada has recently approved six of these products: Alprolix™, an extended half-life factor IX manufactured by Biogen in March 2014; Eloctate® an extended half-life factor VIII manufactured by Biogen in August 2014; Rixubis™, a standard half-life factor IX manufactured by Baxalta in September 2014; Nuwiq® a recombinant FVIII product manufactured by Octapharma using a human cell line in November 2014; Zonovate®, a standard half-life factor VIII manufactured by Novo Nordisk in January 2015; and Kovaltry™, a standard half-life factor VIII manufactured by Bayer in January 2016.

Extended half-life products
The development of extended half-life products is the first major improvement in care for hemophilia since the advent of virally safe concentrates and prophylaxis in the late 1980s.The half-life of factor IX is extended 2.5 to 5 times, factor VIII 1.5 times and recombinant factor VIIa possibly eight-fold. This has the potential to reduce the frequency of infusions, an advance in convenience, or increase the trough levels in prophylaxis, a clear therapeutic advance. Currently, prophylaxis aims to maintain at least a 1% factor level at all times. Should physicians and patients be content with 1% or is a higher trough level desirable to prevent bleeding?

More potent products/Novel mechanisms of action
Some of the products in development are designed to be more potent and more effective in stopping bleeding or to be based on entirely novel mechanisms of action. This is critically important for patients with inhibitors, for whom current treatments are not nearly as effective as conventional treatments for patients with hemophilia A and B without inhibitors.

Broader portfolios for companies
The pipeline sees the development of broader portfolios for companies so that they can market products in all three major areas—hemophilia A, hemophilia B, von Willebrand disease and inhibitors—and not just one or two, as is the case today. This has the potential to increase world supply and competition. In a worldwide market growing by 8% a year, this is a good thing.

While not all of these products will make it to market, many are very promising. We will update these charts periodically. We invite you to consult this page regularly to follow progress.

More details on these clinical trials are available on the Web site of the U.S. National Institutes of Health. Go to www.clinicaltrials.gov and type 'hemophilia' in the search box.


Clinical trials


PRODUCTS IN THE PIPELINE


Factor concentrates

Inhibitors

Non-factor coagulation products

Gene therapy


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The future of care for inherited bleeding disorders

Full interview with Dr. David Lillicrap (November 2017)