(AGENPARL) – LONDON (UNITED KINGDOM), gio 26 marzo 2020
At the time of designation, medicines containing factor VIII were authorised in the EU for the treatment of haemophilia A, to replace the missing protein. However, factor VIII medicines did not work in some patients with haemophilia A because the immune system (the body’s natural defences) can produce ‘inhibitors’ (antibodies) against factor VIII which stop the factor VIII medicine from working. In these cases, other treatments needed to be used, such as factor VIIa (the activated form of factor VII, another protein involved in blood clotting), either alone or as part of a combination treatment.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with haemophilia A because results from early studies showed that the medicine works in patients who have developed inhibitors to authorised treatments. In addition, studies in experimental models suggest that the medicine may be safer than some authorised treatments, because it lowers the risk of clots in blood vessels.
These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.