Altuviiio [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy has been approved by the US Food and Drug Administration (US FDA) to treat a type of inherited bleeding disorder known as Hemophilia A.
The medicine is for routine prophylaxis and on-demand therapy in order to control bleeding episodes, as well as perioperative management (surgery) for adults and children with Hemophilia A. This medication is the first and only treatment for inherited bleeding disorder (Hemophilia A) that mainly delivers normal to almost-normal factor activity levels (beyond 40%) for most of the week with once-weekly dosing.
This treatment significantly reduces bleeding episodes in comparison to prior factor VIII prophylaxis. The simply recommended dosage of medicine Altuviiio is 50 IU/kg, intended for all patients and for different clinical junctures.
What is Hemophilia A:
Hemophilia A, also known as factor VIII (8) deficiency or classic hemophilia, is a rare genetic caused by missing or defective clotting protein named factor VIII (FVIII). In this chronic condition, the ability of an individual`s blood to clot precisely is impaired, leading to immoderate bleeds and connate bleeds into joints that can cause joint destruction and life-long pain, and potentially affect the quality of life (QoL). The severity of the disease is specified by the clotting factor activity level in an individual’s blood, and there is a negative correlation between the probability of bleeding and factor activity levels.
As per US CDC, Hemophilia occurs in roughly 1 in 5,617 live male births. Hemophilia A is 4th times as common as Hemophilia B. This bleeding disorder affects all races and ethnic populations. Although hemophilia A is passed down from parents to their children, approximately 1/3 of cases seen have no known or previous family history.
About Altuviiio:
Altuviiio is a novel VWF (von Willebrand Factor) independent recombinant factor VIII therapy, designed to enhance protection from bleeding episodes with once-weekly prophylactic dosing for both adults and children with hemophilia A. The drug has a 3 o 4 fold longer half-life relative to standard and enhanced half-life factor VIII products. It is the 1st factor VIII therapy that has demonstrated promising activity in order to break through the VWF ceiling, which imposes a half-life limitation on the previous generation factor VIII therapies. The treatment builds on the innovative Fc fusion technology by adding a region of VWF and XTEN polypeptides to enhance its time in circulation.
Reference:
https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-a