FDA Approves Weekly Hemophilia A Therapy

The FDA has approved Altuviiio to treat patients with hemophilia A, a rare bleeding disorder. It occurs in about one in 5,000 male births annually, and more rarely in females. People with hemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Sanofi and Sobi have collaborated on the development and commercialization of the therapy.

Previously referred to as efanesoctocog alfa, Altuviiio is a novel, recombinant factor VIII replacement therapy indicated for the prevention and on-demand treatment to control bleeding episodes in adults and children with hemophilia A. It is designed to extend protection from bleeds with once-weekly prophylactic dosing.

Sanofi officials expect Altuviiio to be available beginning in April 2023. They would not specify a list price until the product is commercially available but a spokesperson said Sanofi will price Altuviiio at parity to the annual cost of treating a prophylaxis patient on Eloctate, another recombinant factor VIII from Sanofi/Sobi to treat patients with hemophilia A. Sanofi provides Eloctate at $2.38 per IU, and company officials would not be more specific. Maryland’s Medicaid system indicated that in 2020, Elocate was one of the top 10 drugs in spending, with $951,166 spent for Eloctate 3,000 unit.

The spokesperson said Sanofi will provide support and resources to patients, including disease and medication education, electronic enrollment, financial assistance, insurance investigation paired with ePrescribing technology, and ongoing support to help address barriers throughout the treatment journey.

Lynn Malec, M.D.

“This approval marks an important clinical advancement for the hemophilia community because we have an option that can achieve higher levels of factor activity with a single simplified weekly dose,” Lynn Malec, M.D., medical director of Comprehensive Center for Bleeding Disorders and associate investigator at The Versiti Blood Research Institute, and associate professor of Medicine and Pediatrics at The Medical College of Wisconsin

The approval was based on the XTEND-1 phase 3 study, which found that Altuviiio demonstrated a clinically meaningful prevention of bleeds in people with severe hemophilia A over a 52-week period. Altuviiio was able to deliver high sustained factor activity levels in the normal to near-normal range for the majority of the week with once weekly prophylaxis dosing. Once-weekly treatment provided significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate of 0.70.Altuviiio met the key secondary endpoint with a significant reduction of 77% in annualized bleeding rates versus prior factor prophylaxis based on an intra-patient comparison.

The therapy was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events were headache, arthralgia, fall, and back pain.