Fidanacogene elaparvovec for haemophilia B

Fidanacogene elaparvovec 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.
Fidanacogene elaparvovec is a medicinal product administered intravenously. Fidanacogene elaparvovec is a gene therapy that delivers a copy of the gene that encodes for factor IX in a patient’s liver cells. Through this, this medicinal product helps to maintain a sustained level factor IX in the blood of adult male with haemophilia B. If licensed, Fidanacogene elaparvovec will offer an additional treatment option for adult males with moderately severe or severe haemophilia B.