First Gene Therapy Treatment for Haemophilia to be Licensed
Written by Jeff Courtney, July 6, 2022
Last week the European Medicines Agency recommended that a license be granted to Roctavian a gene therapy for treating haemophilia A.
Gene therapies for haemophilia offer the chance of a long term and potentially curative treatment to prevent painful bleeds and vastly reduce the need for frequent injections. However, as yet there are no licensed gene therapy treatments for haemophilia and none are currently due to be commissioned by the NHS in any part of the UK.
While these are promising treatments issues remain that need to be overcome. Firstly, the clotting factor level achieved with some gene therapies, particularly those for haemophilia A, appear to drop off over time so we don’t know how long they will last. There is also a lot of variability in the outcomes people get and for some people they don’t work at all. Most people on trials have also seen significant side effects from needing treatment with corticosteroids for six months or more and escalating doses of gene therapy can lead to liver toxicity which can have an impact on factor expression as well as liver health.
Trials have excluded children, and most people with previous inhibitors or ongoing inhibitors, liver damage, HIV infection and/or antibodies to the AAV vector. So, these treatments, even if licensed and commissioned by the NHS will not be available for everyone.
There are more than ten different gene therapies for both haemophilia A and B in the pipeline and we continue to monitor the data closely and cautiously, as with all new treatments.
More data is going to be published at the international haematology conference ISTH this weekend on this and other treatments. If they are shown to be safe and effective, we will work together with NICE and the NHS to make these innovative treatments available to people with bleeding disorders in the UK but for now they are still only on the horizon.