Gene Therapy for Haemophilia B to be licensed next year

Written by Jeff Courtney, December 16, 2022

A gene therapy for haemophilia B has moved a step closer to being licensed for use in the UK. Today, the CHMP has announced a positive opinion for EtranaDez (Etranacogene Dezaparvovec) which will be marketed as Hemgenix. This product was developed by Uniqure and will be marketed by CSL Behring.

From here the European Commission will usually accept the recommendation and a MHRA license for use in Great Britain can then be granted on that basis. We expect this to happen in spring 2022. The license will be for adults with severe or moderately severe (<2%) haemophilia B.

The Haemophilia Society has already been working with NHS England and NICE to consider how this new treatment could be used in England. We are in the process of making submissions to NICE who have scheduled a meeting for July 2023 to make a decision on whether they will recommend it for use in the NHS in England.

We expect similar consideration to take place in Scotland and Wales.

It is difficult to make the case to NICE for expensive new treatments to be used, and a gene therapy for haemophilia B, as very long acting treatment, that might work for a decades, comes with a lot of uncertainty. However, we will be arguing that while there are already effective treatments for managing haemophilia B substantial unmet need remains with treatment outcomes being suboptimal for some people, and there being a very large burden of treatment.

Many people with haemophilia have also told use of the anxiety and worry in managing their condition and how they avoid certain activities and situations. This treatment may offer a good alternative for some people with severe or moderately severe haemophilia B.

We are creating a working group for people with haemophilia B or parents of children with haemophilia B to help us make the case for this treatment and for other improvements in care for people with haemophilia B. If you would like to be involved please email [email protected] to get involved.