The adeno-associated virus (AAV) vector-based therapy is given as a one-shot intravenous infusion, delivering a gene for Factor IX, a clotting factor that is deficient in haemophilia B. In trials, it cut the annualised bleed rate for patients, as well as reducing their need for regularly injected Factor IX replacement therapies.
Hemgenix (etranacogene dezaparvovec) has been cleared for use in adults who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhages, or repeated, serious spontaneous bleeding episodes, said the FDA.
