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ALTUVIIIO (efanesoctocog alfa) is the first Factor VIII replacement therapy to offer bleed protection with once-weekly dosing. In February, Sanofi announced that FDA had approved the drug for routine prophylaxis and on-demand treatment to control bleeding episodes. Its use also extends to perioperative management (surgery) for adults and children with hemophilia A.
Sanofi developed in collaboration with Sobi. Barclays projects that the drug will top €2 billion in peak sales.
Supportive findings from XTEND-kids study
The current FDA approval is supported by interim data from XTEND-Kids. The sBLA seeks to update ALTUVIIIO’s label with the final pediatric results.
In the XTEND-Kids study, 74 patients on weekly ALTUVIIIO prophylaxis experienced a median annual bleed rate of zero. ALTUVIIIO also maintained factor activity levels above 40% for around three days. The safety profile was similar to adults with no concerning side effects.
Traditionally, hemophilia A is treated with routine prophylaxis, which involves regularly infusing patients with clotting factor concentrates to prevent bleeding episodes. This approach is common in individuals with severe hemophilia A while on-demand treatment provides intermittent support to individuals with mild or moderate hemophilia A who have no prior bleeding history.
For patients with the condition, frequency of standard factor replacement can be a major treatment burden, often requiring injections multiple times per week. This can be especially challenging for children.
Traditional Hemophilia A therapies
Hemophilia A is a genetic disorder that affects the blood’s ability to clot properly. The main treatment for severe hemophilia A involves replacing the clotting factor you need through a tube in a vein. This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood.
Other therapies involved to treat the condition include desmopressin, which is used for some forms of mild hemophilia. In addition, emicizumab (Hemlibra) is a newer drug that doesn’t include clotting factors that can help prevent bleeding episodes. Other therapies include clot-preserving medications known as anti-fibrinolytics, fibrin sealants and physical therapy.
Filed Under: Biologics, Hematology, Pediatrics, Regulatory affairs
