Never have so many clotting factor concentrates been in development or “in the pipeline”. So many, in fact, that it has become difficult to keep track of them all. While a lot of these are still at the pre-clinical stage, (when the research is conducted on mice and other animals), many are already in Phase III trials. Phase III trials involve haemophilia patients, and this is the final stage before an application is made to market the product commercially. If the results of the clinical trials are positive, we may begin to see these products come on the market.
The development of longer-acting products could be the first major improvement in care for haemophilia since the advent of virally safe concentrates and prophylaxis in the late 1980’s. 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.
Some of the products in development are designed to be more potent and more effective in stopping bleeding. This is critically important for patients with inhibitors, for whom current treatments are not nearly as effective as conventional treatments for patients with haemophilia A and B without inhibitors.
The pipeline sees the development of broader portfolios for companies so that they can market products in all three major areas (haemophilia A, haemophilia B 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.