Type of medical product:

Protein based

Novelty:

Innovative

Field:

Congenital diseases

Indications for use:

Hemophilia A

Status:

Preclinical trials in vivo

Original therapeutic, GNR-021, is a recombinant long-acting form  of human clotting factor VIII produced with PASylation® technology. GNR-021 contains a B-domain-deleted form of clotting factor VIII with added 200 amino acids PAS-fragment. . GNR-021 is produced using biotechnological methods. . Producing cell strain is obtained from Chinese hamster ovary (CHO) cells. 

Therapeutic action of factor VIII-based agents in hemophilia A patients is related to the restoration of coagulation and hemostasis.. Hemophilia A is a congenital disease (recessive, X-linked one – F8C gene at Xq28 locus of the long arm) caused by insufficiency of active clotting factor VIII in plasma. This disease is the most prevalent hemophilia form with the incidence of 1:5-10000 (of male population). Hemophilia patients develop hemorrhages in joints, muscles and internal organs either spontaneous or trauma- or surgery-induced. Risk of patient death because of bleeding in vital organs is dramatically increased (National Hemophilia Foundation, 2002). Without treatment average life span of such patients is about 30 years with complete disability  onset at 12 years of age. In the Russian Federation there are about 7500 hemophilia patients in need of treatment (according to the World Federation of Hemophilia).

Hemophilia A treatment implies intravenous administration of human factor VIII (FVIII) produced from blood plasma or with recombinant technologies. A considerable constraint for replacement treatment with FVIII in hemophilia A patients is the need for frequent infusions due to a short half life of the agent in blood.  FVIII elimination half-life in patients is 12 to 14 hours. Prophylactic infusions of factor VIII has to be administered as frequently as 3-4 times a week to avoid bleeding episodes. Thus, there is a high need for long-acting FVIII.