announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec).
Hemgenix is the first and only gene therapy approved for haemophilia B. At $3.5m per dose, it is the most expensive single-use gene therapy in the US. The high price tag may be hard to defend, but with lifetime treatment costs for haemophilia B for some, the single-dose treatment could prove far more cost-effective. Despite this, the Institute for Clinical and Economic Review (ICER) claimed that a fairer price would be in the region of $2.9m.
In the past year, Hemgenix has been granted approval by the US Food and Drug Administration (FDA) and has received conditional marketing authorisation from the European Commission (EC) and the UK鈥檚 Medicines and Healthcare products Regulatory Agency (MHRA). This followed positive results from 鈥檚 Phase III HOPE-B trial (NCT03569891). In the study, Hemgenix reduced or eliminated the need for prophylactic treatment in 94% of patients.
, Behring鈥檚 North America general manager and senior vice-president Bob Lojewski said that the company was “thrilled” by this latest milestone and looked forward to more patients benefitting from the therapy.
Hemgenix works by dosing a patient with an engineered adeno-associated virus (AAV), containing the gene responsible for producing a protein called factor IX. This protein helps to form blood clots and stop bleeding, with haemophilia B arising when the body is partially or completely unable to produce it. Once administered, the body will then be able to produce factor IX and prevent severe bleeds.
Already a big player in haemophilia treatments, CSL licensed exclusive global rights to Hemgenix from gene therapy developers back in 2021. that it had received $100m from this first commercial sale milestone, on top of a $450m upfront payment. It is eligible for up to $1.6bn based on future milestones, as well as royalties amounting to more than 20% of net product sales.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataHaemophilia B is a rare condition. It predominantly affects males, with the 33,000 cases in males across the US. Despite the limited market, the price tags of haemophilia make it potentially lucrative.
that the global market for haemophilia A and haemophilia B treatments will grow to $14.7bn by 2030, up from $9.3bn in 2020 鈥� an annual growth of nearly 5%. Gene therapies have long been predicted as the next big thing in haemophilia treatment, and are likely to drive this growth.
GlobalData is the parent company of Pharmaceutical Technology.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
