The first gene therapy drug for the treatment of hemophilia B.

Hemophilia B is an inherited disease that results in a malfunction of the gene that regulates the synthesis of blood coagulation factor IX. As a result, patients suffer from bleeding, which is very difficult to stop. With hemophilia, the risk of death of a patient from a hemorrhage in the brain and other vital organs increases sharply, even with a minor injury.

Currently, hemophilia B is treated with replacement therapy (blood clotting factor IX is administered intravenously, in severe form several times a week). This type of therapy is quite effective but is associated with a large number of inconveniences for patients, and therefore the development of innovative drugs has is a relevant objective.

In 2022, the first gene therapy drug called Hemgenix was registered in the US. This drug is administered by infusion and is designed to replace the gene that codes for factor IX. Hemgenix uses a viral vector to deliver the gene to the liver, where it is taken up by cells and stimulates the production of clotting factor IX.

In the European Union, Hemgenix has been approved based on the results of the HOPE-B study. According to the results of the study, patients with hemophilia B treated with Hemgenix experienced a stable and prolonged increase in the average level of factor IX activity by 36.9 %, which resulted in a 64 % decrease in the adjusted annual bleeding rate.

In the US, Hemgenix costs $3.5 million. In Europe, the therapy could cost around $1 million, but the final price has not yet been announced.

It is worth noting that a gene therapy drug for the treatment of hemophilia B is also being developed in Russia. Research in this area is carried out by the Biocad company. Preclinical studies have now been completed in support of the first phase of clinical trials.

According to the Russian register for medicinal products, in February 2023, the first Phase I-II clinical trial (No. ANB-002-1/SAFRAN “An open-label non-comparative study of the safety, pharmacodynamics and efficacy of ANB-002 in a single dose in increasing doses in patients with hemophilia B”) started. Completion of the study is scheduled for 2031. 54 patients will take part in it.