Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical company Sobi shared that they were working to improve therapeutic options for patients with hemophilia A. Their therapy ALTUVIIIO recently received FDA approval as a routine prophylaxis and on-demand treatment for bleeding episodes; it may also be used for perioperative management. 

What is ALTUVIIIO?

As described in its Prescribing Information, ALTUVIIIO is:

a recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A.

ALTUVIIIO is not indicated for the treatment of von Willebrand disease. Given its long half-life compared to other Factor VIII products, ALTUVIIIO only requires dosing once per week and helps to sustain Factor VIII levels over 40%. 

ALTUVIIIO has received Fast Track, Orphan Drug, and Breakthrough Therapy designations in the United States, as well as Orphan Drug designation in the European Union. In the United States, it was evaluated under Priority Review. Common side effects relating to ALTUVIIIO treatment include back and joint pain, as well as headache. This treatment will most likely be available in the US by some point in April 2023. 

Research Data

The recent approval hinged, in part, to data from the Phase 3 XTEND-1 trial. 159 individuals aged 12+ with severe hemophilia A enrolled. They were split into two treatment groups: prophylaxis and on-demand. The study found that ALTUVIIIO improved factor VIII activity, reduced the risk of bleeds, and lowered annual bleeding rate. ALTUVIIIO was also safe and well-tolerated. Additional data from the Phase 3 XTEND-Kids trial found that ALTUVIIIO also seemed safe and effective in children younger than 12 years old. 

The Basics of Hemophilia A

Also known as classic hemophilia or factor VIII deficiency, hemophilia A is an inherited bleeding disorder caused by defective or lacking factor VIII, a clotting protein. It results from a mutation on the X chromosome. While hemophilia A is most often inherited, this gene mutation is spontaneous in an estimated 30% of cases. Females living with hemophilia A are rare; this condition predominantly impacts males. The severity of hemophilia A varies from patient to patient. Approximately 60% of individuals affected are on the severe end of the disorder.

Symptoms relating to hemophilia A may include:

• Frequent and/or prolonged nosebleeds or bleeding from the gums
• Easy bruising
• Joint bleeds (as well as associated pain and inflammation)
• Limited range of mobility in the affected joints
• Hematomas under the skin
• Hematuria (blood in the urine)
• Dark and bloody stool 
• Bleeding after cuts, trauma, dental work, or surgery
• Excessive menstruation and bleeding after childbirth (in females) 

Outside of ALTUVIIIO, treatments for hemophilia A include Hemgenix, Hemlibra, DDAVP (Desmopressin Acetate), and concentrated FVIII.