Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments.
Hemophilia is a rare bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding following an injury or surgery. In severe cases, heavy bleeding occurs after minor trauma or in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The major types of this disorder are hemophilia A and hemophilia B. Although the two types have very similar signs and symptoms, they are caused by genetic changes in different genes.
XTEND-ed Study
Altuviiio is a high-sustained, factor VIII therapy designed to extend bleeding protection for adults and children with hemophilia A.
The phase 3 XTEND-ed study is a multicenter, open-label, three-arm, clinical trial testing the long-term safety and efficacy of Altuviiio in adult and pediatric patients. Through analyses, it was observed that the treatment was highly effective in preventing bleeding. This leads to improvement and maintenance of joint health over the two year period. Safety profile was also consistent with that observed in initial studies, with no new adverse events.
ATLAS Study
Fitusiran is a first-in-class antithrombin-lowering therapy for the potential prophylactic treatment for patients with hemophilia A or B, regardless of inhibitor status. The therapy has been developed to prevent bleeding and lower antithrombin.
The phase 3 ATLAS study is a clinical trial testing the safety and efficacy of fitusiran in patients with hemophilia. It was observed that fitusiran prophylaxis under an antithrombin-based dosing regimen was successful in mitigating the risk of thrombotic events. It also reduced elevated liver enzymes and inflammation of the gallbladder or gallstones. The therapy’s safety profile was favorable and results show safety in major surgeries in patients on fitusiran. These results reinforce those presented in prior studies.
A regulatory submission for fitusiran is in front of the U.S. Food and Drug Administration with a target action date of March 28, 2025. To learn more, click here.
For more information on hemophilia and other rare hematologic diseases, visit https://checkrare.com/diseases/hematologic-disorders/
