Etranacogene dezaparvovec is in development for the treatment of moderately severe or severe haemophilia B in adult males. Haemophilia B is a hereditary bleeding disorder that results in the blood taking longer to clot than normal. The disorder is caused by having a faulty version of the F9 gene. The F9 gene provides instructions for making a protein called coagulation factor IX which is released following injury to a blood vessel to form a clot and prevent further blood loss. A faulty F9 gene results in insufficient production of functional clotting factor protein IX. In severe cases, this can result in spontaneous bleeding into the joints, muscles or brain causing serious complications.
Etranacogene dezaparvovec is given to patients by intravenous injection. It works by using a harmless viral vector to insert a highly functional copy of the F9 gene. This means that the body can make more functional factor IX clotting protein so the blood does not take as long to clot following an injury and there is no spontaneous bleeding into the joints, muscles or brain. If licensed, etranacogene dezaparvovec will offer an additional treatment option for adult males with moderately severe or severe haemophilia B.