Phase 3 of Fitusiran, an all-around tx for hemophilia

Multinational clinical, it is conducted on five patients in three places, including Severance Hospital

Phase 3 clinical trials of Fitusiran, a treatment candidate substance that is effective for both hemophilia A and B, are also being conducted in Korea.

 

The Ministry of Food and Drug Safety approved a phase 3 clinical trial of Sanofi-Aventis Korea on the 18th.

 

This clinical trial is a phase 3, single group, multi-organ, multinational clinical trial to investigate the efficacy and safety of Fitusiran prevention therapy in male severe type A or type B test subjects over the age of 12 with or without inhibitory antibodies to coagulation factor VIII or IX.

 

In phase 3 of multinational clinical trials targeting 75 male hemophilia patients around the world, Korea has been approved for clinical trials in five patients.

 

Clinical trials are conducted at Kangdong Kyung Hee University Hospital, Severance Hospital, and Inha University Hospital.

 

The Fitusiran global clinical trial was voluntarily suspended by Sanofi in October 2020 after non-fatal symptoms of thrombosis were found in phase 3 clinical trial participants.

 

Sanofi also stopped all studies in September 2017 when one patient died of thromboembolism in the brain during phase 2 clinical trials.

 

Hemophilia is a congenital hemorrhagic disease caused by a lack of blood clotting factors, and the incidence rate is estimated to be around one per 10,000 people.

 

Depending on the type of insufficient coagulation factors, it is divided into two types: hemophilia A and hemophilia B, with type A accounting for 80% of the total and type B accounting for the remaining 20%.

 

Fitusiran is a small interference RNA (siRNA) treatment that targets antithrombin, which is a treatment for hemophilia type A and type B patients and promotes thrombin production by lowering the level of antithrombin to prevent hemophilia's hemostasis and bleeding.

 

Currently, Hemlibra, which is used for hemophilia type A, ranks first in the hemophilia treatment market, and Sanofi and Novonordisk's Concizumab are the development treatments used for both hemophilia type A and type B patients.